Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain
Open accessAbstract
Applications of RNA interference for neuroscience research have been limited by a lack of simple and efficient methods to deliver oligonucleotides to primary neurons in culture and to the brain. Here, we show that primary neurons rapidly internalize hydrophobically modified siRNAs (hsiRNAs) added directly to the culture medium without lipid formulation. We identify functional hsiRNAs targeting the mRNA of huntingtin, the mutation of which is responsible for Huntington's disease, and show that direct uptake in neurons induces potent and specific silencing in vitro. Moreover, a single injection of unformulated hsiRNA into mouse brain silences Htt mRNA with minimal neuronal toxicity. Thus, hsiRNAs embody a class of therapeutic oligonucleotides that enable simple and straightforward functional studies of genes involved in neuronal biology and neurodegenerative disorders in a native biological context.
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Such conjugated siRNAs are retained near the brain injection site, even with continuous monthly administration.
“However, they are retained near the brain injection site, even with continuous monthly administration4, 7.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
The Results section on mouse-brain distribution (page not provided) reports a steep fluorescence gradient from the injection site 24 hours after a single injection, with no fluorescence visually detectable on the opposite side. This supports limited distribution after one injection, but not retention during continuous monthly administration; the Materials and methods section describes single injections, not a monthly regimen. (LLM, conf=0.88)
Experimental — accuracy not yet measuredIBA-1 and GFAP are well-established markers of immune stimulation.
“To evaluate the impact of Di-siRNA injection on microglia activation and gliosis, we measured two well-established markers of immune stimulation, IBA-1 and GFAP.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
The Results section (page not provided) uses IBA-1-positive microglia to assess innate immune activation and says IBA-1 is upregulated after brain injury. The cited paper’s passages do not identify GFAP as a marker or use it to assess gliosis, so they support only the IBA-1 part of the claim. (LLM, conf=0.86)
Experimental — accuracy not yet measuredDARPP32 protein expression is an established marker for medium spiny neurons in the striatum.
“To assess neuronal viability, we measured DARPP32 protein expression, an established marker for medium spiny neurons in the striatum7.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
The Results section (page unavailable) calls DARPP-32 “an established marker for striatal neuron viability,” and Materials and methods describes counting DARPP-32-positive neurons in the striatum. Our copy does not identify those neurons specifically as medium spiny neurons, so that part of the claim is not supported. (LLM, conf=0.84)
Experimental — accuracy not yet measuredConjugating cholesterol to an siRNA generates steep concentration gradients around CSF-brain interfaces at the expense of delivery to deeper brain structures.
“conjugating cholesterol to an siRNA promotes robust cellular association, but generates steep concentration gradients around CSF–brain interfaces at the expense of delivery to deeper brains structures [11].”
From Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid (2025) · cites this paper as [11]
The Results section (‘hsiRNA distribution in vivo in mouse brain after intrastriatal injection’; page not provided) reports a steep fluorescence gradient from the injection site, with no visually detectable fluorescence on the opposite side. The paper does not place that gradient at a CSF–brain interface or establish that cholesterol conjugation caused reduced delivery to deeper brain structures. (LLM, conf=0.86)
Experimental — accuracy not yet measuredHtt is a disease-relevant target that is ubiquitously expressed by CNS cells, and a well-validated siRNA sequence exists for it.
“we chose Htt as a disease-relevant target that is ubiquitously expressed by CNS cells and for which a well-validated siRNA sequence exists (“Htt10150”) [11].”
From Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid (2025) · cites this paper as [11]
The Introduction identifies Htt as the causative gene in Huntington’s disease. The Results (‘Potent and specific silencing with unformulated hsiRNAs in primary neurons’; page unavailable) show that HTT10150 silences Htt in primary neurons, and the in vivo Results report dose-dependent silencing in mouse brain. These passages support the disease relevance and validated siRNA sequence, but do not establish that Htt is ubiquitously expressed by CNS cells. (LLM, conf=0.87)
Experimental — accuracy not yet measured
Cholesterol conjugation to siRNA promotes robust cellular association but generates steep concentration gradients around CSF-brain interfaces instead of achieving more homogeneous distribution within the brain.
“However, cellular uptake can also counter transport; for example, cholesterol to an siRNA promotes robust cellular association, but generates steep concentration gradients around CSF-brain interfaces instead of achieving more homogeneous distribution within the brain.25”
From Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous system (2024) · cites this paper as [25]
The Results sections on neuronal internalization and in vivo distribution report that cholesterol-conjugated hsiRNA associates with cells and that fluorescence forms a steep gradient after injection into the mouse striatum. The gradient emanated from the injection site, not a CSF–brain interface. The paper does not establish that cholesterol conjugation caused the limited distribution or compare it with a more homogeneously distributed siRNA. (LLM, conf=0.88)
Experimental — accuracy not yet measuredHtt is a disease-relevant target that is ubiquitously expressed by CNS cells and for which a well-validated siRNA sequence exists.
“We chose Htt as a disease-relevant target that is ubiquitously expressed by CNS cells and for which a well-validated siRNA sequence exists (“Htt10150”).25”
From Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous system (2024) · cites this paper as [25]
The Introduction identifies Htt as the causative gene in Huntington’s disease, and the Results report that HTT10150 silences Htt mRNA in mouse brain. Those passages support the disease relevance of the target and the efficacy of the named siRNA, but our copy does not establish that Htt is ubiquitously expressed by CNS cells. (LLM, conf=0.86)
Experimental — accuracy not yet measuredFully chemically-stabilized siRNAs conjugated to hydrophobic ligands like cholesterol, DHA, or DHA with a phosphocholine head group are successfully internalized by brain cells.
“When fully chemically-stabilized siRNAs are conjugated to hydrophobic ligands like cholesterol7, docosahexaenoic acid (DHA)8, or DHA with a phosphocholine head group10, they are successfully internalized by brain cells11.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
siRNAs are large, negatively-charged, double-stranded molecules that require formulation or chemical conjugation for delivery to tissues, especially the CNS.
“siRNAs are large, negatively-charged, double-stranded molecules that require formulation6 or chemical conjugation7–10 for delivery to tissues (especially CNS).”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
IBA-1 is located within a major histocompatibility complex and increases as a result of microglial activation.
“IBA-1 is located within a major histocompatibility complex and increases as a result of microglial activation7.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
Conjugating cholesterol to an siRNA promotes robust cellular association.
“conjugating cholesterol to an siRNA promotes robust cellular association, but generates steep concentration gradients around CSF–brain interfaces at the expense of delivery to deeper brains structures [11].”
From Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid (2025) · cites this paper as [11]
Di-siRNAHTT is perfectly complementary to both human HTT and mouse Htt genes.
“Di-siRNAHTT is perfectly complementary to both human HTT and mouse Htt genes7.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [7]
The Materials and methods describe hsiRNAs designed against human huntingtin, and the Results report that HTT10150 silences mouse Htt. Neither section identifies Di-siRNAHTT or states that it is perfectly complementary to both human and mouse targets (searched: Di-siRNAHTT, perfect complementarity, cross-species sequence matching). (LLM, conf=0.76)
Experimental — accuracy not yet measured
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Percentages, sums and reported differences re-computed from the paper’s own numbers.
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Cites
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Fire, A., Xu, S., Montgomery, M. K., Kostas, S. A., Driver, S. E., & Mello, C. C. (1998) · Nature · doi:10.1038/35888 · Cited by 15,429 · see citing works
Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes
Alvarez-Erviti, L., Seow, Y., Yin, H., Betts, C., Lakhal, S., & Wood, M. J. A. (2011) · Nature Biotechnology · doi:10.1038/nbt.1807 · Cited by 4,930 · see citing works
Cellular Scaling Rules for the Brains of Marsupials: Not as “Primitive” as Expected
Santos, S. E. D., Porfírio, J., Cunha, F., Manger, P. R., Tavares, W. C., Pessôa, L. M., Raghanti, M. A., Sherwood, C. C., & Herculano‐Houzel, S. (2017) · Brain Behavior and Evolution · doi:10.1159/000452856 · Cited by 2,651 · see citing works
Functional siRNAs and miRNAs exhibit strand bias
Khvorova, A., Reynolds, A., & Jayasena, S. D. (2003) · Cell · doi:10.1016/s0092-8674(03)00801-8 · Cited by 2,645 · see citing works
Self-citationNeuN, a neuronal specific nuclear protein in vertebratesxs
Mullen, R. J., Buck, C. R., & Smith, A. M. (1992) · Development · doi:10.1242/dev.116.1.201 · Cited by 2,351 · see citing works
Asymmetry in the Assembly of the RNAi Enzyme Complex
Schwarz, D. S., Hutvágner, G., Du, T., Xu, Z., Aronin, N., & Zamore, P. D. (2003) · Cell · doi:10.1016/s0092-8674(03)00759-1 · Cited by 2,323 · see citing works
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Soutschek, J., Akinc, A., Bramlage, B., Charissé, K., Constien, R., Donoghue, M., Elbashir, S. M., Geick, A., Hadwiger, P., Harborth, J., et al. (2004) · Nature · doi:10.1038/nature03121 · Cited by 2,189 · see citing works
Rational siRNA design for RNA interference
Reynolds, A., Leake, D., Boese, Q., Scaringe, S., Marshall, W. S., & Khvorova, A. (2004) · Nature Biotechnology · doi:10.1038/nbt936 · Cited by 2,094 · see citing works
Self-citationTransvascular delivery of small interfering RNA to the central nervous system
Kumar, P., Wu, H., McBride, J. L., Jung, K.-E., Hee Kim, M., Davidson, B. L., Kyung Lee, S., Shankar, P., & Manjunath, N. (2007) · Nature · doi:10.1038/nature05901 · Cited by 1,246 · see citing works
The promises and pitfalls of RNA-interference-based therapeutics
Castanotto, D., & Rossi, J. J. (2009) · Nature · doi:10.1038/nature07758 · Cited by 1,219 · see citing works
Imai, Y., Ibata, I., Ito, D., Ohsawa, K., & Kohsaka, S. (1996) · Biochemical and Biophysical Research Communications · doi:10.1006/bbrc.1996.1112 · Cited by 872 · see citing works
Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides
Geary, R. S., Norris, D. A., Yu, R. Z., & Bennett, C. F. (2015) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.01.008 · Cited by 846 · see citing works
Kordasiewicz, H. B., Stanek, L. M., Wancewicz, E. V., Mazur, C., McAlonis, M. M., Pytel, K. A., Artates, J. W., Weiss, A., Cheng, S. H., Shihabuddin, L. S., et al. (2012) · Neuron · doi:10.1016/j.neuron.2012.05.009 · Cited by 770 · see citing works
Current prospects for RNA interference-based therapies
Davidson, B. L., & McCray, P. B. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2968 · Cited by 760 · see citing works
Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons
DiFiglia, M., Sapp, E., Chase, K., Schwarz, C., Meloni, A., Young, C., Martin, E., Vonsattel, J.-P., Carraway, R., Reeves, S. A., et al. (1995) · Neuron · doi:10.1016/0896-6273(95)90346-1 · Cited by 749 · see citing works
Self-citationOuimet, C. C., Miller, P. E., Hemmings, H. C., Walaas, S. I., & Greengard, P. (1984) · Journal of Neuroscience · doi:10.1523/jneurosci.04-01-00111.1984 · Cited by 655 · see citing works
Bartlett, D. W. (2006) · Nucleic Acids Research · doi:10.1093/nar/gkj439 · Cited by 593 · see citing works
Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing
Deleavey, G. F., & Damha, M. J. (2012) · Chemistry & Biology · doi:10.1016/j.chembiol.2012.07.011 · Cited by 590 · see citing works
Design of Noninflammatory Synthetic siRNA Mediating Potent Gene Silencing in Vivo
Judge, A. D., Bola, G., Lee, A. C. H., & MacLachlan, I. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.002 · Cited by 571 · see citing works
Inflammatory Neurodegeneration and Mechanisms of Microglial Killing of Neurons
Brown, G. C., & Neher, J. J. (2010) · Molecular Neurobiology · doi:10.1007/s12035-010-8105-9 · Cited by 526 · see citing works
Microglia Biology in Health and Disease
Garden, G. A., & Möller, T. (2006) · Journal of Neuroimmune Pharmacology · doi:10.1007/s11481-006-9015-5 · Cited by 494 · see citing works
Silencing disease genes in the laboratory and the clinic
Watts, J. K., & Corey, D. R. (2011) · The Journal of Pathology · doi:10.1002/path.2993 · Cited by 472 · see citing works
Chemically modified siRNA: tools and applications
Watts, J. K., Deleavey, G. F., & Damha, M. J. (2008) · Drug Discovery Today · doi:10.1016/j.drudis.2008.05.007 · Cited by 431 · see citing works
DiFiglia, M., Sena‐Esteves, M., Chase, K. O., Sapp, E., Pfister, E., Sass, M., Yoder, J., Reeves, P., Pandey, R. K., Rajeev, K. G., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0708285104 · Cited by 417 · see citing works
Self-citationRNA interference: listening to the sound of silence.
Zamore, P. D. (2001) · Nature Structural Biology · doi:10.1038/nsb0901-746 · Cited by 368 · see citing works
Activation of the mammalian immune system by siRNAs
Marques, J. T., & Williams, B. R. G. (2005) · Nature Biotechnology · doi:10.1038/nbt1161 · Cited by 357 · see citing works
Huntington's disease gene (IT15) is widely expressed in human and rat tissues
Li, S.-H., Schilling, G., Young, W. S., Li, X., Margolis, R. L., Stine, O. C., Wagster, M. V., Abbott, M. H., Franz, M. L., Ranen, N. G., et al. (1993) · Neuron · doi:10.1016/0896-6273(93)90127-d · Cited by 347 · see citing works
Boudreau, R. L., McBride, J. L., Martins, I., Shen, S., Xing, Y., Carter, B. J., & Davidson, B. L. (2009) · Molecular Therapy · doi:10.1038/mt.2009.17 · Cited by 339 · see citing works
Shitaka, Y., Tran, H., Bennett, R. E., Sanchez, L., Levy, M. A., Dikranian, K. T., & Brody, D. L. (2011) · Journal of Neuropathology & Experimental Neurology · doi:10.1097/nen.0b013e31821f891f · Cited by 307 · see citing works
Carroll, J. B., Warby, S. C., Southwell, A. L., Doty, C. N., Greenlee, S., Skotte, N. H., Hung, G., Bennett, C. F., Freier, S. M., & Hayden, M. R. (2011) · Molecular Therapy · doi:10.1038/mt.2011.201 · Cited by 294 · see citing works
Developmental and cell type‐specific expression of the neuronal marker NeuN in the murine cerebellum
Weyer, A., & Schilling, K. (2003) · Journal of Neuroscience Research · doi:10.1002/jnr.10655 · Cited by 266 · see citing works
Song, E., Lee, S., Dykxhoorn, D. M., Novina, C. D., Zhang, D., Crawford, K. D., Černý, J., Sharp, P. A., Lieberman, J., Manjunath, N., et al. (2003) · Journal of Virology · doi:10.1128/jvi.77.13.7174-7181.2003 · Cited by 230 · see citing works
Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting
de Paula, D., Bentley, M. V. L. B., & Mahato, R. I. (2007) · RNA · doi:10.1261/rna.459807 · Cited by 227 · see citing works
A protocol for designing siRNAs with high functionality and specificity
Birmingham, A., Anderson, E. M., Sullivan, K., Reynolds, A., Boese, Q., Leake, D., Karpilow, J. M., & Khvorova, A. (2007) · Nature Protocols · doi:10.1038/nprot.2007.278 · Cited by 222 · see citing works
Self-citationThakker, D. R., Natt, F., Hüsken, D., Maier, R., Müller, M., van der Putten, H., Hoyer, D., & Cryan, J. F. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0406214101 · Cited by 211 · see citing works
Transfection Techniques for Neuronal Cells: Table 1.
Karra, D., & Dahm, R. (2010) · The Journal of Neuroscience · doi:10.1523/jneurosci.0183-10.2010 · Cited by 209 · see citing works
FedExosomes: Engineering Therapeutic Biological Nanoparticles that Truly Deliver
Marcus, M., & Leonard, J. N. (2013) · Pharmaceuticals · doi:10.3390/ph6050659 · Cited by 201 · see citing works
Use of adeno-associated viral vector for delivery of small interfering RNA
Tomar, R. S., Matta, H., & Chaudhary, P. M. (2003) · Oncogene · doi:10.1038/sj.onc.1206733 · Cited by 197 · see citing works
Southwell, A. L., Skotte, N. H., Kordasiewicz, H. B., Østergaard, M. E., Watt, A. T., Carroll, J. B., Doty, C. N., Villanueva, E. B., Petoukhov, E., Vaid, K., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.153 · Cited by 144 · see citing works
Oligonucleotide therapeutic approaches for Huntington disease
Sah, D. W. Y., & D, N. A. M. (2011) · Journal of Clinical Investigation · doi:10.1172/jci45130 · Cited by 132 · see citing works
Masotti, A., Mossa, G., Cametti, C., Ortaggi, G., Bianco, A., Grosso, N. D., Malizia, D., & Esposito, C. (2008) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2008.09.017 · Cited by 130 · see citing works
Focused ultrasound for targeted delivery of siRNA and efficient knockdown of Htt expression
Burgess, A., Huang, Y., Querbes, W., Sah, D. W. Y., & Hynynen, K. (2012) · Journal of Controlled Release · doi:10.1016/j.jconrel.2012.08.012 · Cited by 118 · see citing works
Southwell, A. L., Smith, S. E. P., Davis, T. R., Caron, N. S., Villanueva, E. B., Xie, Y., Collins, J. A., Ye, M. L., Sturrock, A., Leavitt, B. R., et al. (2015) · Scientific Reports · doi:10.1038/srep12166 · Cited by 109 · see citing works
Cytotoxicity issues pertinent to lipoplex-mediated gene therapy in-vivo
Dass, C. R. (2002) · Journal of Pharmacy and Pharmacology · doi:10.1211/0022357021778817 · Cited by 97 · see citing works
Widespread suppression of huntingtin with convection-enhanced delivery of siRNA
Stiles, D. K., Zhang, Z., Ge, P., Nelson, B. D., Grondin, R., Ai, Y., Hardy, P., Nelson, P. T., Guzaev, A. P., Butt, M. T., et al. (2011) · Experimental Neurology · doi:10.1016/j.expneurol.2011.11.020 · Cited by 91 · see citing works
Dolga, A. M., Granic, I., Blank, T., Knaus, H., Spiess, J., Luiten, P. G. M., Eisel, U. L. M., & Nijholt, I. M. (2008) · Journal of Neurochemistry · doi:10.1111/j.1471-4159.2008.05701.x · Cited by 84 · see citing works
Byrne, M., Tzekov, R. T., Wang, Y., Rodgers, A., Cardia, J. P., Ford, G., Holton, K., Pandarinathan, L., Lapierre, J., Stanney, W., et al. (2013) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2013.0148 · Cited by 78 · see citing works
Self-citationNonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7
Ramachandran, P. S., Boudreau, R. L., Schaefer, K. A., Spada, A. R. L., & Davidson, B. L. (2014) · Molecular Therapy · doi:10.1038/mt.2014.108 · Cited by 60 · see citing works
Zou, L., Huang, L., Hayes, R. L., Black, C., Qiu, Y. H., Perez-Polo, J. R., Le, W., Clifton, G. L., & Yang, K. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300936 · Cited by 59 · see citing works
Comprehensive evaluation of canonical versus Dicer-substrate siRNA in vitro and in vivo
Foster, D. J., Barros, S. A., Duncan, R., Shaikh, S., Cantley, W. L., Dell, A., Bulgakova, E., O′Shea, J., Taneja, N., Kuchimanchi, S., et al. (2012) · RNA · doi:10.1261/rna.031120.111 · Cited by 42 · see citing works
Age-Dependent Microglial Activation in Immature Brains After Hypoxia- Ischemia
Ferrazzano, P. A., Chanana, V., Uluç, K., Fidan, E., Aktüre, E., Kintner, D. B., Cengiz, P., & Sun, D. (2013) · CNS & Neurological Disorders - Drug Targets · doi:10.2174/1871527311312030007 · Cited by 42 · see citing works
Huo, H., Gao, Y., Wang, Y., Zhang, J., Wang, Z., Jiang, T., & Wang, S. (2015) · Journal of Colloid and Interface Science · doi:10.1016/j.jcis.2015.01.043 · Cited by 40 · see citing works
Liposomal transfection efficiency and toxicity on glioma cell lines
Bell, H. S., Kimber, W. L., Li, M., & Whittle, I. R. (1998) · Neuroreport · doi:10.1097/00001756-199803300-00005 · Cited by 34 · see citing works
Identifying siRNA-Induced Off-Targets by Microarray Analysis
Anderson, E. M., Boese, Q., Khvorova, A., & Karpilow, J. M. (2008) · Methods in molecular biology · doi:10.1007/978-1-59745-191-8_4 · Cited by 34 · see citing works
Self-citationConstruction of shRNA lentiviral vector
Song, H., & Yang, P. (2010) · North American Journal of Medical Sciences · doi:10.4297/najms.2010.2598 · Cited by 22 · see citing works
RNA Interference in Neuroscience: Progress and Challenges
Miller, V. M., Paulson, H. L., & Gonzalez‐Alegre, P. (2005) · Cellular and Molecular Neurobiology · doi:10.1007/s10571-005-8447-4 · Cited by 17 · see citing works
Koshinaga, M., Suma, T., Fukushima, M., Tsuboi, I., Aizawa, S., & Katayama, Y. (2007) · PubMed · doi:10.14670/hh-22.129 · Cited by 15 · see citing works
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Research works printed but not linked: 2
- Karra, D and Dahm, R (2010). Transfection techniques for neuronal cells. J Neurosci 30: 6171–6177. 20445041 PMC6632737
- Byrne, M, Tzekov, R, Wang, Y, Rodgers, A, Cardia, J, Ford, G et al. (2013). Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye. J Ocul Pharmacol Ther 29: 855–864. 24180627
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The chemical evolution of oligonucleotide therapies of clinical utility
Khvorova, A., & Watts, J. K. (2017) · Nature Biotechnology · doi:10.1038/nbt.3765 · Cited by 1,216 · see citing works
Self-citationHaraszti, R. A., Miller, R., Stoppato, M., Sere, Y. Y., Coles, A., Didiot, M.-C., Wollacott, R., Sapp, E., Dubuke, M. L., Li, X., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.09.015 · Cited by 567 · see citing works
Self-citationExosome-mediated delivery of hydrophobically modified siRNA for huntingtin mRNA silencing
Didiot, M.-C., Hall, L. M., Coles, A. H., Haraszti, R. A., Godinho, B. M., Chase, K., Sapp, E., Ly, S., Alterman, J. F., Hassler, M. R., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.126 · Cited by 483 · see citing works
Self-citationTabrizi, S. J., Estevez‐Fraga, C., van Roon‐Mom, W., Flower, M. D., Scahill, R. I., Wild, E. J., Muñoz-Sanjuán, I., Sampaio, C., Rosser, A., & Leavitt, B. R. (2022) · The Lancet Neurology · doi:10.1016/s1474-4422(22)00121-1 · Cited by 339 · see citing works
Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates
Brown, K. M., Nair, J. K., Janas, M. M., Anglero-Rodriguez, Y. I., Dang, L. T. H., Peng, H., Theile, C. S., Castellanos-Rizaldos, E., Brown, C., Foster, D., et al. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01334-x · Cited by 258 · see citing works
Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology · doi:10.1038/s41587-019-0205-0 · Cited by 227 · see citing works
Self-citationCurrent Development of siRNA Bioconjugates: From Research to the Clinic
Chernikov, I. V., Vlassov, V. V., & Chernolovskaya, E. L. (2019) · Frontiers in Pharmacology · doi:10.3389/fphar.2019.00444 · Cited by 216 · see citing works
RNAi modulation of placental sFLT1 for the treatment of preeclampsia
Turanov, A. A., Lo, A. S., Hassler, M. R., Makris, A., Ashar-Patel, A., Alterman, J. F., Coles, A. H., Haraszti, R. Á., Roux, L., Godinho, B. M. D. C., et al. (2018) · Nature Biotechnology · doi:10.1038/nbt.4297 · Cited by 212 · see citing works
Self-citationDiverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo
Biscans, A., Coles, A. H., Haraszti, R. Á., Echeverria, D., Hassler, M. R., Osborn, M. F., & Khvorova, A. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky1239 · Cited by 206 · see citing works
Self-citationFunctional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles
O’Loughlin, A. J., Mäger, I., de Jong, O. G., Varela, M. A., Schiffelers, R. M., Andaloussi, S. E., Wood, M. J. A., & Vader, P. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.021 · Cited by 200 · see citing works
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo
Hassler, M. R., Turanov, A. A., Alterman, J. F., Haraszti, R. A., Coles, A. H., Osborn, M. F., Echeverria, D., Nikan, M., Salomon, W. E., Roux, L., et al. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky037 · Cited by 181 · see citing works
Self-citationOsborn, M. F., Coles, A. H., Biscans, A., Haraszti, R. Á., Roux, L., Davis, S. M., Ly, S., Echeverria, D., Hassler, M. R., Godinho, B. M. D. C., et al. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky1232 · Cited by 147 · see citing works
Self-citationImproving siRNA Delivery <i>In Vivo</i> Through Lipid Conjugation
Osborn, M. F., & Khvorova, A. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0725 · Cited by 137 · see citing works
Self-citationVisualization of self-delivering hydrophobically modified siRNA cellular internalization
Ly, S., Navaroli, D. M., Didiot, M.-C., Cardia, J., Pandarinathan, L., Alterman, J. F., Fogarty, K., Standley, C., Lifshitz, L. M., Bellve, K. D., et al. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkw1005 · Cited by 132 · see citing works
Self-citation5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo
Haraszti, R. Á., Roux, L., Coles, A. H., Turanov, A. A., Alterman, J. F., Echeverria, D., Godinho, B. M. D. C., D, N. A. M., & Khvorova, A. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx507 · Cited by 128 · see citing works
Self-citationNucleic acid drug vectors for diagnosis and treatment of brain diseases
Lü, Z., Shen, J., Yang, H. J., Wang, J., Zhao, R., Zhang, T.-L., Guo, J. R., & Zhang, X. (2023) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-01298-z · Cited by 108 · see citing works
Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles
Haraszti, R. Á., Miller, R., Didiot, M.-C., Biscans, A., Alterman, J. F., Hassler, M. R., Roux, L., Echeverria, D., Sapp, E., DiFiglia, M., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.05.024 · Cited by 106 · see citing works
Self-citationHaraszti, R. Á., Miller, R., Dubuke, M. L., Rockwell, H. E., Coles, A. H., Sapp, E., Didiot, M.-C., Echeverria, D., Stoppato, M., Sere, Y. Y., et al. (2019) · iScience · doi:10.1016/j.isci.2019.05.029 · Cited by 103 · see citing works
Self-citationSava, V., Fihurka, O., Khvorova, A., & Sanchez-Ramos, J. (2019) · Nanomedicine: Nanotechnology, Biology and Medicine · doi:10.1016/j.nano.2019.102119 · Cited by 83 · see citing works
Self-citationOral delivery of non-viral nucleic acid-based therapeutics - do we have the guts for this?
O’Driscoll, C. M., Bernkop‐Schnürch, A., Friedl, J. D., Véronique Préat, A., & Jannin, V. (2019) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2019.03.027 · Cited by 95 · see citing works
Nikan, M., Osborn, M. F., Coles, A. H., Godinho, B. M., Hall, L. M., Haraszti, R. A., Hassler, M. R., Echeverria, D., Aronin, N., & Khvorova, A. (2016) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2016.50 · Cited by 85 · see citing works
Self-citationClinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases
Holm, A., Hansen, S. N., Klitgaard, H., & Kauppinen, S. (2022) · RNA Biology · doi:10.1080/15476286.2022.2066334 · Cited by 83 · see citing works
Emerging Roles of Exosomes in Huntington’s Disease
Ananbeh, H., Vodička, P., & Skalníková, H. K. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22084085 · Cited by 82 · see citing works
Grijalvo, S., Alagia, A., Jorge, A. F., & Eritja, R. (2018) · Genes · doi:10.3390/genes9020074 · Cited by 77 · see citing works
Mehta, A., Michler, T., & Merkel, O. M. (2021) · Advanced Healthcare Materials · doi:10.1002/adhm.202001650 · Cited by 74 · see citing works
Biscans, A., Caiazzi, J., Davis, S. M., McHugh, N., Sousa, J., & Khvorova, A. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa595 · Cited by 68 · see citing works
Self-citationShmushkovich, T., Monopoli, K. R., Homsy, D., Leyfer, D., Betancur-Boissel, M., Khvorova, A., & Wolfson, A. D. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky745 · Cited by 72 · see citing works
Self-citationNuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin
Didiot, M.-C., Ferguson, C. M., Ly, S., Coles, A. H., Smith, A. O., Bicknell, A. A., Hall, L. M., Sapp, E., Echeverria, D., Pai, A. A., et al. (2018) · Cell Reports · doi:10.1016/j.celrep.2018.07.106 · Cited by 64 · see citing works
Self-citationBiscans, A., Coles, A. H., Echeverria, D., & Khvorova, A. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.03.028 · Cited by 64 · see citing works
Self-citationGodinho, B. M. D. C., Henninger, N., Bouley, J., Alterman, J. F., Haraszti, R. Á., Gilbert, J. W., Sapp, E., Coles, A. H., Biscans, A., Nikan, M., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.08.005 · Cited by 62 · see citing works
Self-citationO’Reilly, D., Belgrad, J., Ferguson, C. M., Summers, A., Sapp, E., McHugh, C. A., Mathews, E. W., Boudi, A., Buchwald, J. E., Ly, S., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.05.006 · Cited by 60 · see citing works
Self-citationChemical optimization of siRNA for safe and efficient silencing of placental sFLT1
Davis, S. M., Hariharan, V. N., Lo, A. S., Turanov, A. A., Echeverria, D., Sousa, J., McHugh, N., Biscans, A., Alterman, J. F., Karumanchi, S. A., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.06.009 · Cited by 59 · see citing works
Self-citationEnhancing siRNA efficacy in vivo with extended nucleic acid backbones
Yamada, K., Hariharan, V. N., Caiazzi, J., Miller, R., Ferguson, C. M., Sapp, E., Fakih, H. H., Tang, Q., Yamada, N., Furgal, R. C., et al. (2024) · Nature Biotechnology · doi:10.1038/s41587-024-02336-7 · Cited by 59 · see citing works
Self-citationSegal, M., Biscans, A., Gilles, M.-E., Anastasiadou, E., Luca, R. D., Lim, J., Khvorova, A., & Slack, F. J. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.11.008 · Cited by 58 · see citing works
Self-citationChitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain
Sanchez‐Ramos, J., Song, S., Kong, X., Foroutan, P., Martinez, G. V., Dominguez-Viqueria, W., Mohapatra, S. S., Mohapatra, S., Haraszti, R. Á., Khvorova, A., et al. (2017) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2017.11.013 · Cited by 57 · see citing works
Self-citationMolecularly engineered siRNA conjugates for tumor-targeted RNAi therapy
Lee, J. W., Choi, J., Choi, Y., Kim, K., Yang, Y., Kim, S. H., Yoon, H. Y., & Kwon, I. C. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.09.040 · Cited by 55 · see citing works
Recent preclinical and clinical advances in oligonucleotide conjugates
Craig, K. P., Abrams, M. T., & Amiji, M. M. (2018) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2018.1473375 · Cited by 56 · see citing works
Biscans, A., Haraszti, R. Á., Echeverria, D., Miller, R., Didiot, M.-C., Nikan, M., Roux, L., D, N. A. M., & Khvorova, A. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.03.019 · Cited by 55 · see citing works
Self-citationLigtenberg, M. A., de Coaña, Y. P., Shmushkovich, T., Yoshimoto, Y., Truxová, I., Yang, Y., Betancur-Boissel, M., Eliseev, A. V., Wolfson, A. D., & Kiessling, R. W. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.04.015 · Cited by 53 · see citing works
Godinho, B. M. D. C., Gilbert, J. W., Haraszti, R. A., Coles, A. H., Biscans, A., Roux, L., Nikan, M., Echeverria, D., Hassler, M., & Khvorova, A. (2017) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0690 · Cited by 52 · see citing works
Self-citationDelivery of Oligonucleotides: Efficiency with Lipid Conjugation and Clinical Outcome
Tran, P., Weldemichael, T., Liu, Z., & Li, H. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14020342 · Cited by 51 · see citing works
Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo
Biscans, A., Ly, S., McHugh, N., Cooper, D., & Khvorova, A. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.07.041 · Cited by 50 · see citing works
Self-citationConroy, F., Miller, R., Alterman, J. F., Hassler, M. R., Echeverria, D., Godinho, B. M. D. C., Knox, E. G., Sapp, E., Sousa, J., Yamada, K., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-33061-x · Cited by 50 · see citing works
Self-citationDivalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection
Hariharan, V. N., Shin, M., Chang, C., O’Reilly, D., Biscans, A., Yamada, K., Guo, Z., Somasundaran, M., Tang, Q., Monopoli, K. R., et al. (2023) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2219523120 · Cited by 48 · see citing works
Self-citationYenilmez, B., Wetoska, N., Kelly, M., Echeverria, D., Min, K., Lifshitz, L., Alterman, J. F., Hassler, M. R., Hildebrand, S., DiMarzio, C., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2021.11.007 · Cited by 46 · see citing works
Self-citationStructural Modifications of siRNA Improve Its Performance In Vivo
Chernikov, I. V., Ponomareva, U. A., & Chernolovskaya, E. L. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24020956 · Cited by 44 · see citing works
Chemical modulation of siRNA lipophilicity for efficient delivery
Tai, W. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.06.022 · Cited by 44 · see citing works
McAdam, R. L., Morton, A., Gordon, S. L., Alterman, J. F., Khvorova, A., Cousin, M. A., & Smillie, K. J. (2019) · Neurobiology of Disease · doi:10.1016/j.nbd.2019.104637 · Cited by 44 · see citing works
Self-citationLy, S., Echeverria, D., Sousa, J., & Khvorova, A. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.07.029 · Cited by 38 · see citing works
Self-citationMiller, J. B., & Siegwart, D. J. (2018) · Nano Research · doi:10.1007/s12274-018-2099-4 · Cited by 40 · see citing works
Synthesis and biological properties of triazole-linked locked nucleic acid
Sharma, V. K., Singh, S. K., Krishnamurthy, P. M., Alterman, J. F., Haraszti, R. Á., Khvorova, A., Prasad, A. K., & Watts, J. K. (2017) · Chemical Communications · doi:10.1039/c7cc04092j · Cited by 39 · see citing works
Self-citationColes, A. H., Osborn, M. F., Alterman, J. F., Turanov, A. A., Godinho, B. M. D. C., Kennington, L. A., Chase, K. O., D, N. A. M., & Khvorova, A. (2015) · Nucleic Acid Therapeutics · doi:10.1089/nat.2015.0578 · Cited by 38 · see citing works
Self-citationBortolozzi, A., Manashirov, S., Chen, A., & Artigas, F. (2021) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2021.107873 · Cited by 37 · see citing works
Non-viral siRNA delivery to T cells: Challenges and opportunities in cancer immunotherapy
Hoeck, J. V., Braeckmans, K., Smedt, S. C. D., & Raemdonck, K. (2022) · Biomaterials · doi:10.1016/j.biomaterials.2022.121510 · Cited by 37 · see citing works
Nikan, M., Osborn, M. F., Coles, A. H., Biscans, A., Godinho, B. M. D. C., Haraszti, R. A., Sapp, E., Echeverria, D., DiFiglia, M., Aronin, N., et al. (2017) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.7b00226 · Cited by 35 · see citing works
Self-citationDevelopment of nucleic acid medicines based on chemical technology
Kawamoto, Y., Wu, Y., Takahashi, Y., & Takakura, Y. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114872 · Cited by 34 · see citing works
Fàbrega, C., Aviñó, A. M., Navarro, N., Jorge, A. F., Grijalvo, S., & Eritja, R. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15020320 · Cited by 33 · see citing works
Therapeutic Potential of Nucleic Acids when Combined with Extracellular Vesicles
Jurgielewicz, B. J., Stice, S. L., & Yao, Y. (2021) · Aging and Disease · doi:10.14336/ad.2021.0708 · Cited by 32 · see citing works
Sun, M., Qin, F., Bu, Q., Zhao, Y., Yang, X., Zhang, D., & Cen, X. (2025) · MedComm · doi:10.1002/mco2.70386 · Cited by 33 · see citing works
Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington’s Disease
Tousley, A. R., Iuliano, M., Weisman, E., Sapp, E., Zhang, N., Vodička, P., Alexander, J. S., Aviolat, H., Gatune, L. W., Reeves, P., et al. (2018) · Journal of Huntington s Disease · doi:10.3233/jhd-180311 · Cited by 28 · see citing works
Self-citationAntisense therapies in neurological diseases
de Courssou, J.-B. B., Dürr, A., Adams, D., Corvol, J., & Mariani, L. (2021) · Brain · doi:10.1093/brain/awab423 · Cited by 28 · see citing works
Ferguson, C. M., Godinho, B. M. D. C., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.152203 · Cited by 27 · see citing works
Self-citationPendergraff, H. M., Krishnamurthy, P. M., Debacker, A. J., Moazami, M. P., Sharma, V. K., Niitsoo, L., Yu, Y., Tan, Y. N., Haitchi, H. M., & Watts, J. K. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.012 · Cited by 25 · see citing works
Systematic analysis of siRNA and mRNA features impacting fully chemically modified siRNA efficacy
Davis, S. M., Hildebrand, S. R., MacMillan, H. J., Monopoli, K. R., Buchwald, J. E., Sousa, J., Cooper, D., Ly, S., Echeverria, D., McHugh, N., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf479 · Cited by 25 · see citing works
Self-citationGene Therapies for Polyglutamine Diseases
Matos, C. A., Carmona, V., Vijayakumar, U. G., Lopes, S., Albuquerque, P., Conceição, M., Nobre, R. J., Nóbrega, C., & de Almeida, L. P. (2018) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-71779-1_20 · Cited by 25 · see citing works
Chemical strategies for strand selection in short-interfering RNAs
Varley, A., & Desaulniers, J. (2021) · RSC Advances · doi:10.1039/d0ra07747j · Cited by 25 · see citing works
Maguregui, A., & Abe, H. (2020) · ChemBioChem · doi:10.1002/cbic.202000009 · Cited by 24 · see citing works
Davis, S. M., Sousa, J., Vangjeli, L., Hassler, M. R., Echeverria, D., Knox, E. G., Turanov, A. A., Alterman, J. F., & Khvorova, A. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.05.010 · Cited by 23 · see citing works
Self-citationPapadopoulou, A. S., Gomez-Paredes, C., Mason, M. A., Taxy, B. A., Howland, D., & Bates, G. P. (2019) · Scientific Reports · doi:10.1038/s41598-019-52411-2 · Cited by 23 · see citing works
Revisiting gene delivery to the brain: silencing and editing
Conniot, J., Talebian, S., Simões, S. M. N., Ferreira, L., & Conde, J. (2020) · Biomaterials Science · doi:10.1039/d0bm01278e · Cited by 23 · see citing works
Zhang, S., Yang, Y., Lv, X., Zhou, X., Zhao, W., Meng, L., Zhu, S., Zhang, Z., & Wang, Y. (2024) · Brain Sciences · doi:10.3390/brainsci14111049 · Cited by 21 · see citing works
Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs
Osborn, M. F., Coles, A. H., Golebiowski, D., Echeverria, D., Moazami, M. P., Watts, J. K., Sena‐Esteves, M., & Khvorova, A. (2018) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-17-1144 · Cited by 20 · see citing works
Self-citationDendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity
Fakih, H. H., Tang, Q., Summers, A., Shin, M., Buchwald, J. E., Gagnon, R., Hariharan, V. N., Echeverria, D., Cooper, D., Watts, J. K., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.102080 · Cited by 20 · see citing works
Self-citationLoading of Extracellular Vesicles with Hydrophobically Modified siRNAs
Didiot, M.-C., Haraszti, R. Á., D, N. A. M., & Khvorova, A. (2018) · Methods in molecular biology · doi:10.1007/978-1-4939-7652-2_16 · Cited by 20 · see citing works
Self-citationYamada, K., Hildebrand, S. R., Davis, S. M., Miller, R., Conroy, F., Sapp, E., Caiazzi, J., Alterman, J. F., Roux, L., Echeverria, D., et al. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab1126 · Cited by 18 · see citing works
Self-citationSelf-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo
Zhang, H., Kelly, K. L., Lee, J. L. C., Echeverria, D., Cooper, D., Panwala, R., Amrani, N., Chen, Z., Gaston, N., Wagh, A. A., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad1125 · Cited by 19 · see citing works
Self-citationSarli, S. L., Fakih, H. H., Kelly, K., Devi, G., Rembetsy-Brown, J. M., McEachern, H. R., Ferguson, C. M., Echeverria, D., Lee, J., Sousa, J., et al. (2024) · Nucleic Acids Research · doi:10.1093/nar/gkae260 · Cited by 18 · see citing works
Self-citationUSP10 Targeted Self-Deliverable siRNA to Prevent Scarring in the Cornea
Boumil, E. F., Castro, N., Phillips, A. T., Chatterton, J. E., McCauley, S. M., Wolfson, A. D., Shmushkovich, T., Ridilla, M., & Bernstein, A. M. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.07.032 · Cited by 17 · see citing works
Cheng, S.-Y., Caiazzi, J., Biscans, A., Alterman, J. F., Echeverria, D., McHugh, N., Hassler, M. R., Jolly, S. M., Giguere, D., Cipi, J., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.102088 · Cited by 19 · see citing works
Self-citationEnhancing Neuronal Cell Uptake of Therapeutic Nucleic Acids with Tetrahedral DNA Nanostructures
Martins, A. S. G., Reis, S., Benson, E., Domingues, M. M., Cortinhas, J., Silva, J. A. V., Santos, S. D., Santos, N. C., Pêgo, A. P., & Moreno, P. M. D. (2024) · Small · doi:10.1002/smll.202309140 · Cited by 17 · see citing works
Iron Oxide Nanoparticle-Mediated siRNA Delivery System for Huntington’s Disease Treatment
Rohiwal, S. S., Nguyen, T. D., Kamenná, E., Klı́ma, J., Vaškovičová, M., Sekac, D., Šlouf, M., Pavlová, E., Stepanek, P., Babuka, D., et al. (2023) · ACS Applied Nano Materials · doi:10.1021/acsanm.2c03936 · Cited by 16 · see citing works
Gene Therapy: The View from NCATS
Brooks, P. J., Yang, N. N., & Austin, C. P. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.29018.pjb · Cited by 16 · see citing works
Ochocinska, M. J., Zloković, B. V., Searson, P. C., Crowder, A. T., Kraig, R. P., Ljubimova, J. Y., Mainprize, T. G., Banks, W. A., Warren, R. Q., Kindzelski, A., et al. (2017) · Fluids and Barriers of the CNS · doi:10.1186/s12987-017-0061-6 · Cited by 16 · see citing works
Vlaho, D., Fakhoury, J. F., & Damha, M. J. (2017) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0702 · Cited by 16 · see citing works
Zhu, Y., Wang, F., Xia, Y., Wang, L., Lin, H., Zhong, T., & Wang, X. (2024) · Reviews in the Neurosciences · doi:10.1515/revneuro-2024-0043 · Cited by 15 · see citing works
Development of stable liquid formulations for oligonucleotides
Poecheim, J., Graeser, K. A., Hoernschemeyer, J., Becker, G., Storch, K., & Printz, M. (2018) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2018.05.029 · Cited by 15 · see citing works
Romanò, R., Luca, M. D., Fiore, V. S. D., Pecoraro, M., Lattante, S., Sabatelli, M., Bella, V. L., & Bucci, C. (2022) · Brain Communications · doi:10.1093/braincomms/fcac315 · Cited by 14 · see citing works
Mathews, E. W., Coffey, S. R., Gärtner, A., Belgrad, J., Bragg, R. M., O’Reilly, D., Cantle, J. P., McHugh, C. A., Summers, A., Fentz, J., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-64936-4 · Cited by 14 · see citing works
Self-citationTherapeutic potential of extracellular vesicles in neurodegenerative disorders
Roudi, S., Rädler, J., & Andaloussi, S. E. (2023) · Handbook of clinical neurology · doi:10.1016/b978-0-323-85555-6.00017-5 · Cited by 14 · see citing works
Fihurka, O., Sanchez‐Ramos, J., & Sava, V. (2018) · PubMed · doi:10.29011/2577-1477.100055 · Cited by 13 · see citing works
Blevins, M. S., Chin, S., Wang, J., Famili, A., Cadang, L., Wei, B., & Zhang, K. (2025) · Analytical Chemistry · doi:10.1021/acs.analchem.4c05968 · Cited by 12 · see citing works
Cholesterol Conjugates of Small Interfering RNA: Linkers and Patterns of Modification
Chernikov, I. V., Ponomareva, U. A., Meschaninova, M. I., Bachkova, I. K., Vlassov, V. V., Аркадьевна, З. М., & Chernolovskaya, E. L. (2024) · Molecules · doi:10.3390/molecules29040786 · Cited by 11 · see citing works
Cell membrane-coated nanoparticles for neurodegenerative disorders management
Thakur, N., Kumar, T., Singh, C., Kumar, A., & Kumar, A. (2025) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2025.125875 · Cited by 11 · see citing works
A Review On Huntington Protein: Insight Into Protein Aggregation and Therapeutic Interventions
Ekambaram, S., Ram, V., & Rajasekaran, R. (2022) · Current Drug Metabolism · doi:10.2174/1389200223666220321103942 · Cited by 11 · see citing works
Important Aspects of siRNA Design for Optimal Efficacy In Vitro and In Vivo
Bhakta-Yadav, M. S., & Brown, T. L. (2025) · International Journal of Cell Biology · doi:10.1155/ijcb/6663816 · Cited by 10 · see citing works
Chemical Modifications and Design Influence the Potency of Huntingtin Anti-Gene Oligonucleotides
Saher, O., Zaghloul, E. M., Umek, T., Hagey, D. W., Mozafari, N., Danielsen, M. B., Gouda, A. S., Lundin, K. E., Jørgensen, P. T., Wengel, J., et al. (2023) · Nucleic Acid Therapeutics · doi:10.1089/nat.2022.0046 · Cited by 10 · see citing works
Allen, S. E., O’Reilly, D., Miller, R., Sapp, E., Summers, A., Paquette, J. D., Echeverria, D., Bramato, B. M., McHugh, N., Yamada, K., et al. (2024) · Nucleic Acid Therapeutics · doi:10.1089/nat.2024.0027 · Cited by 10 · see citing works
Self-citationHaraszti, R. Á., Coles, A. H., D, N. A. M., Khvorova, A., & Didiot, M.-C. (2017) · BIO-PROTOCOL · doi:10.21769/bioprotoc.2338 · Cited by 10 · see citing works
Self-citationIdentification of selective and non-selective C9ORF72 targeting in vivo active siRNAs
Gilbert, J. W., Kennedy, Z., Godinho, B. M. D. C., Summers, A., Weiss, A., Echeverria, D., Bramato, B. M., McHugh, N., Cooper, D., Yamada, K., et al. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102291 · Cited by 9 · see citing works
Self-citationFihurka, O., Aradi, S. D., Sava, V., & Sanchez‐Ramos, J. (2023) · Journal of Nanotechnology and Nanomaterials · doi:10.33696/nanotechnol.4.043 · Cited by 8 · see citing works
- Authors
- Alterman, J. F., Hall, L. M., Coles, A. H., Hassler, M. R., Didiot, M.-C., Chase, K., Abraham, J., Sottosanti, E., Johnson, E., Sapp, E., Osborn, M. F., Difiglia, M., Aronin, N., & Khvorova, A.
- Affiliations
- University of Massachusetts Chan Medical School (US)
- Massachusetts General Hospital (US)
- Corresponding author
- Khvorova, Anastasia
- ORCID
- Venue
- Molecular Therapy - Nucleic Acids
- Publisher
- Elsevier BV
- Published
- 2015
- Volume
- 4
- Pages
- e266
- ISSN
- 2162-2531 (ISSN-L)
- PMC
- PMC5014532
- OpenAlex
- W2295697145
- Cited by
- 158
- References
- 58
- Citations by year
- 2026: 11 · 2025: 20 · 2024: 16 · 2023: 17 · 2022: 20 · 2021: 11 · 2020: 10 · 2019: 11 · 2018: 22 · 2017: 13 · 2016: 5 · 2015: 1 · 2013: 1
- FWCI
- 5.94
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Nucleic Acids (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- eScholarship@UMassChan (submittedVersion, cc-by)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- National Institutes of Health (UH2TR000888, 1s10od020012-01, UH3TR000888)
- National Institute of General Medical Sciences (R01GM108803)
- National Center for Advancing Translational Sciences
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › DNA and Nucleic Acid Chemistry
- Keywords
- siRNA delivery, primary neurons, RNA interference, Huntington's disease, oligonucleotide uptake, intracerebroventricular administration, neurodegenerative diseases, gene silencing
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