Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65
Open accessAbstract
Adeno-associated viral vectors (AAVs) have demonstrated potential in applications for neurologic disorders, and the discovery that some AAVs can cross the blood-brain barrier (BBB) after intravenous injection has further expanded these opportunities for non-invasive brain delivery. Anc80L65, a novel AAV capsid designed from in silico reconstruction of the viral evolutionary lineage, has previously demonstrated robust transduction capabilities after local delivery in various tissues such as liver, retina, or cochlea, compared with conventional AAVs. Here, we compared the transduction efficacy of Anc80L65 with conventional AAV9 in the CNS after intravenous, intracerebroventricular (i.c.v.), or intraparenchymal injections. Anc80L65 was more potent at targeting the brain and spinal cord after intravenous injection than AAV9, and mostly transduced astrocytes and a wide range of neuronal subpopulations. Although the efficacy of Anc80L65 and AAV9 is similar after direct intraparenchymal injection in the striatum, Anc80L65's diffusion throughout the CNS was more extensive than AAV9 after i.c.v. infusion, leading to widespread EGFP expression in the cerebellum. These findings demonstrate that Anc80L65 is a highly efficient gene transfer vector for the murine CNS. Systemic injection of Anc80L65 leads to notable expression in the CNS that does not rely on a self-complementary genome. These data warrant further testing in larger animal models.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Later studies characterized Anc80L65, demonstrating great transduction efficiency for retinas, inner ear, and notable expression in the CNS after systemic gene transfer.
“Later, more studies further characterized the vector Anc80L65, demonstrating great transduction efficiency for retinas [71], inner ear [72], and even notable expression in the CNS after systemic gene transfer [73].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [73]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381 · see citing works
Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31868-8 · Cited by 1,952 · see citing works
Chan, K. Y., Jang, M. J., Yoo, B. B., Greenbaum, A., Ravi, N., Wu, W.-L., Sánchez-Guardado, L., Lois, C., Mazmanian, S. K., Deverman, B. E., et al. (2017) · Nature Neuroscience · doi:10.1038/nn.4593 · Cited by 1,578 · see citing works
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520 · see citing works
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456 · see citing works
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273 · see citing works
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
Deverman, B. E., Pravdo, P. L., Simpson, B. P., Kumar, S. R., Chan, K. Y., Banerjee, A., Wu, W.-L., Yang, B., Huber, N., Pasca, S. P., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3440 · Cited by 1,061 · see citing works
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
Mingozzi, F., & High, K. A. (2013) · Blood · doi:10.1182/blood-2013-01-306647 · Cited by 955 · see citing works
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses
Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J., & Wilson, J. M. (2009) · The Journal of Infectious Diseases · doi:10.1086/595830 · Cited by 808 · see citing works
Self-citationIn vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9
Swiech, L., Heidenreich, M., Banerjee, A., Habib, N., Li, Y., Trombetta, J., Sur, M., & Zhang, F. (2015) · Nature Biotechnology · doi:10.1038/nbt.3055 · Cited by 772 · see citing works
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708538 · Cited by 762 · see citing works
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Rangarajan, S., Walsh, L., Lester, W., Perry, D., Madan, B., Laffan, M., Yu, H., Vettermann, C., Pierce, G. F., Wong, W. Y., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708483 · Cited by 694 · see citing works
Immune responses to gene therapy vectors: influence on vector function and effector mechanisms
Bessis, N., Garcia-Cozar, F. J., & Boissier, M. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302364 · Cited by 636 · see citing works
A Neural Circuit for Memory Specificity and Generalization
Xu, W., & Südhof, T. C. (2013) · Science · doi:10.1126/science.1229534 · Cited by 634 · see citing works
Antibody-based protection against HIV infection by vectored immunoprophylaxis
Balazs, A. B., Chen, J., Hong, C. M., Rao, D. S., Yang, L., & Baltimore, D. (2012) · Nature · doi:10.1038/nature10660 · Cited by 545 · see citing works
Self-complementary AAV vectors; advances and applications
McCarty, D. M. (2008) · Molecular Therapy · doi:10.1038/mt.2008.171 · Cited by 515 · see citing works
Duque, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A.-M., Fyfe, J., Moullier, P., Colle, M.-A., & Barkats, M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.71 · Cited by 511 · see citing works
Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501 · see citing works
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
Nault, J.-C., Datta, S., Imbeaud, S., Franconi, A., Mallet, M., Couchy, G., Letouzé, E., Pilati, C., Verret, B., Blanc, J.-F., et al. (2015) · Nature Genetics · doi:10.1038/ng.3389 · Cited by 489 · see citing works
New recombinant serotypes of AAV vectors
Gao, G., Vandenberghe, L., & Wilson, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054065057 · Cited by 487 · see citing works
Self-citationIntegration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors
McCarty, D. M., Young, S. M., & Samulski, R. J. (2004) · Annual Review of Genetics · doi:10.1146/annurev.genet.37.110801.143717 · Cited by 450 · see citing works
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
Hordeaux, J., Wang, Q., Katz, N., Buza, E. L., Bell, P., & Wilson, J. M. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.01.018 · Cited by 426 · see citing works
Improved tools for the Brainbow toolbox
Cai, D., COHEN, K. B., Luo, T., Lichtman, J. W., & Sanes, J. R. (2013) · Nature Methods · doi:10.1038/nmeth.2450 · Cited by 420 · see citing works
Wang, Z., Ma, H.-I., Li, J., Sun, L., Zhang, J., & Xiao, X. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302133 · Cited by 413 · see citing works
Bevan, A. K., Duque, S., Foust, K. D., Morales, P. R., Braun, L., Schmelzer, L., Chan, C. M., McCrate, M., Chicoine, L. G., Coley, B. D., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.157 · Cited by 337 · see citing works
Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale
Lock, M. W., Alvira, M. R., Vandenberghe, L. H., Samanta, A., Toelen, J., Debyser, Z., & Wilson, J. M. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.055 · Cited by 328 · see citing works
Self-citationA synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear
Landegger, L. D., Pan, B., Askew, C., Wassmer, S. J., Gluck, S. D., Galvin, A., Taylor, R., Forge, A., Stankovic, K. M., Holt, J. R., et al. (2017) · Nature Biotechnology · doi:10.1038/nbt.3781 · Cited by 325 · see citing works
Self-citationJohnson, P. R., Schnepp, B. C., Zhang, J., Connell, M. J., Greene, S. M., Yuste, E., Desrosiers, R. C., & Clark, K. R. (2009) · Nature Medicine · doi:10.1038/nm.1967 · Cited by 310 · see citing works
In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector
Zinn, E., Pacouret, S., Khaychuk, V., Turunen, H. T., Carvalho, L. S., Andres-Mateos, E., Shah, S., Shelke, R., Maurer, A. C., Plovie, E., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.019 · Cited by 310 · see citing works
Self-citationLong-term follow-up after gene therapy for canavan disease
Leone, P., Shera, D., McPhee, S. W. J., Francis, J. S., Kolodny, E. H., Bilaniuk, L. T., Wang, D.-J., Assadi, M., Goldfarb, O., Goldman, H. W., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003454 · Cited by 307 · see citing works
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
Samaranch, L., Salegio, E. A., San Sebastian, W., Kells, A. P., Foust, K. D., Bringas, J. R., Lamarre, C., Forsayeth, J., Kaspar, B. K., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.200 · Cited by 299 · see citing works
Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c
Pan, B., Askew, C., Galvin, A., Heman‐Ackah, S. E., Asai, Y., Indzhykulian, A. A., Jodelka, F. M., Hastings, M. L., Lentz, J. J., Vandenberghe, L. H., et al. (2017) · Nature Biotechnology · doi:10.1038/nbt.3801 · Cited by 295 · see citing works
Self-citationZhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.98 · Cited by 291 · see citing works
Meyer, K., Ferraiuolo, L., Schmelzer, L., Braun, L., McGovern, V., Likhite, S., Michels, O., Govoni, A., Fitzgerald, J., Morales, P., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2014.210 · Cited by 286 · see citing works
Assessing the potential for AAV vector genotoxicity in a murine model
Li, H., Malani, N., Hamilton, S. R., Schlachterman, A., Bussadori, G., Edmonson, S. A., Shah, R., Arruda, V. R., Mingozzi, F., Wright, J. F., et al. (2010) · Blood · doi:10.1182/blood-2010-08-302729 · Cited by 234 · see citing works
Serrano-Pozo, A., Muzikansky, A., Gómez-Isla, T., Growdon, J. H., Betensky, R. A., Frosch, M. P., & Hyman, B. T. (2013) · Journal of Neuropathology & Experimental Neurology · doi:10.1097/nen.0b013e3182933788 · Cited by 226 · see citing works
Self-citationProgress toward Gene Therapy for Duchenne Muscular Dystrophy
Chamberlain, J. S., & Chamberlain, J. S. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.02.019 · Cited by 224 · see citing works
Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer
Pulicherla, N., Shen, S., Yadav, S. S., Debbink, K. M., Lakshmanan Govindasamy, A., Agbandje‐McKenna, M., & Asokan, A. (2011) · Molecular Therapy · doi:10.1038/mt.2011.22 · Cited by 223 · see citing works
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Haurigot, V., Marcó, S., Ribera, A., Garcia, M., Ruzo, A., Villacampa, P., Ayuso, E., Añor, S., Andaluz, A., Pineda, M., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci66778 · Cited by 215 · see citing works
Immune Responses to Adeno-Associated Virus Vectors
Zaiss, A. K., & Muruve, D. A. (2005) · Current Gene Therapy · doi:10.2174/1566523054065039 · Cited by 214 · see citing works
Yang, B., Li, S., Wang, H., Guo, Y., Gessler, D. J., Cao, C., Su, Q., Kramer, J., Zhong, L., Ahmed, S. S., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.68 · Cited by 213 · see citing works
Foust, K. D., Salazar, D. L., Likhite, S. B., Ferraiuolo, L., Ditsworth, D., Ilieva, H. S., Meyer, K., Schmelzer, L., Braun, L., Cleveland, D. W., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.211 · Cited by 205 · see citing works
Hudry, E., Dam, D. V., Kulik, W., Deyn, P. P. D., Stet, F. S., Ahouansou, O., Benraiss, A., Delacourte, A., Bougneres, P. F., Aubourg, P. R., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.175 · Cited by 196 · see citing works
Self-citationAdeno-associated virus effectively mediates conditional gene modification in the brain
Kaspar, B. K., Vissel, B., Bengoechea, T. G., Crone, S. A., Randolph‐Moore, L., Müller, R., Brandon, E. P., Schaffer, D. V., Verma, I. M., Lee, K., et al. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.042678699 · Cited by 195 · see citing works
Fu, H., DiRosario, J., Killedar, S., Zaraspe, K., & McCarty, D. M. (2011) · Molecular Therapy · doi:10.1038/mt.2011.34 · Cited by 193 · see citing works
Viral vectors for therapy of neurologic diseases
Choudhury, S. R., Hudry, E., Maguire, C. A., Sena-Esteves, M., Breakefield, X. O., & Grandi, P. (2017) · Neuropharmacology · doi:10.1016/j.neuropharm.2016.02.013 · Cited by 190 · see citing works
Self-citationHudry, E., Dashkoff, J., Roe, A. D., Takeda, S., Koffie, R. M., Hashimoto, T., Scheel, M., Spires-Jones, T., Arbel-Ornath, M., Betensky, R., et al. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3007000 · Cited by 187 · see citing works
Self-citationshRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model
Zharikov, A., Cannon, J. R., Tapias, V., Bai, Q., Horowitz, M. P., Shah, V., Ayadi, A. E., Hastings, T. G., Greenamyre, J. T., & Burton, E. A. (2015) · Journal of Clinical Investigation · doi:10.1172/jci64502 · Cited by 184 · see citing works
Suzuki, J., Hashimoto, K., Xiao, R., Vandenberghe, L. H., & Liberman, M. C. (2017) · Scientific Reports · doi:10.1038/srep45524 · Cited by 175 · see citing works
Self-citationExosome-associated AAV vector as a robust and convenient neuroscience tool
Hudry, E., Martin, C., Gandhi, S., György, B., Scheffer, D. I., Mu, D., Merkel, S. F., Mingozzi, F., Fitzpatrick, Z., Dimant, H., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.11 · Cited by 146 · see citing works
Self-citationMolecular Profiling of Neurons Based on Connectivity
Ekstrand, M. I., Nectow, A. R., Knight, Z. A., Latcha, K. N., Pomeranz, L. E., & Friedman, J. M. (2014) · Cell · doi:10.1016/j.cell.2014.03.059 · Cited by 137 · see citing works
Katz, M. L., Tecedor, L., Chen, Y., Williamson, B. G., Lysenko, E., Wininger, F. A., Young, W. M., Johnson, G. C., Whiting, R. E. H., Coates, J. R., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aac6191 · Cited by 134 · see citing works
Merkel, S. F., Andrews, A. M., Lutton, E. M., Mu, D., Hudry, E., Hyman, B. T., Maguire, C. A., & Ramirez, S. H. (2017) · Journal of Neurochemistry · doi:10.1111/jnc.13861 · Cited by 132 · see citing works
Self-citationBroad protection against influenza infection by vectored immunoprophylaxis in mice
Balazs, A. B., Bloom, J. D., Hong, C. M., Rao, D. S., & Baltimore, D. (2013) · Nature Biotechnology · doi:10.1038/nbt.2618 · Cited by 127 · see citing works
Adeno-associated virus: fit to serve
Zinn, E. M., & Vandenberghe, L. H. (2014) · Current Opinion in Virology · doi:10.1016/j.coviro.2014.07.008 · Cited by 122 · see citing works
Self-citationAAV transcytosis through barrier epithelia and endothelium
Di Pasquale, G., & Chiorini, J. A. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.007 · Cited by 121 · see citing works
Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications
Gaj, T., Epstein, B. E., & Schaffer, D. V. (2015) · Molecular Therapy · doi:10.1038/mt.2015.151 · Cited by 117 · see citing works
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
Choudhury, S., Fitzpatrick, Z., Harris, A. F., Maitland, S. A., Ferreira, J. S., Zhang, Y., Ma, S., Sharma, R. B., Gray‐Edwards, H. L., Johnson, J. A., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.84 · Cited by 115 · see citing works
Self-citationLimberis, M. P., Adam, V. S., Wong, G., Gren, J., Kobasa, D., Ross, T. M., Kobinger, G. P., Tretiakova, A. P., & Wilson, J. M. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3006299 · Cited by 112 · see citing works
Choudhury, S., Harris, A. F., Cabral, D. J., Keeler, A. M., Sapp, E., Ferreira, J. S., Gray‐Edwards, H. L., Johnson, J. A., Johnson, A. K., Su, Q., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.231 · Cited by 110 · see citing works
Wu, J., Zhao, W., Zhong, L., Han, Z., Li, B., Ma, W., Weigel-Kelley, K. A., Warrington, K. H., & Srivastava, A. (2007) · Human Gene Therapy · doi:10.1089/hum.2006.088 · Cited by 108 · see citing works
Koo, T., Popplewell, L., Athanasopoulos, T., & Dickson, G. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.164 · Cited by 104 · see citing works
Ojala, D. S., Sun, S., Santiago-Ortiz, J. L., Shapiro, M. G., Romero, P. A., & Schaffer, D. V. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.006 · Cited by 97 · see citing works
Rosas, L. E., Grieves, J. L., Zaraspe, K., Perle, K. M. D. L., Fu, H., & McCarty, D. M. (2012) · Molecular Therapy · doi:10.1038/mt.2012.197 · Cited by 95 · see citing works
AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease
Passini, M. A., Macauley, S. L., Huff, M. R., Taksir, T. V., Bu, J., Wu, I., Piepenhagen, P. A., Dodge, J. C., Shihabuddin, L. S., O’Riordan, C. R., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.01.011 · Cited by 90 · see citing works
Sevin, C., Benraiss, A., Dam, D. V., Bonnin, D., Nagels, G., Vérot, L., Laurendeau, I., Vidaud, M., Gieselmann, V., Vanier, M., et al. (2006) · Human Molecular Genetics · doi:10.1093/hmg/ddi425 · Cited by 87 · see citing works
Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy
Gong, Y., Mu, D., Prabhakar, S., Moser, A., Musolino, P., Ren, J., Breakefield, X. O., Maguire, C. A., & Eichler, F. S. (2015) · Molecular Therapy · doi:10.1038/mt.2015.6 · Cited by 74 · see citing works
Self-citationKotchey, N. M., Adachi, K., Zahid, M., Inagaki, K., Charan, R. A., Parker, R. S., & Nakai, H. (2011) · Molecular Therapy · doi:10.1038/mt.2011.3 · Cited by 73 · see citing works
Cardiac gene therapy with adeno-associated virus-based vectors
Chamberlain, K., Riyad, J. M., & Weber, T. (2017) · Current Opinion in Cardiology · doi:10.1097/hco.0000000000000386 · Cited by 73 · see citing works
Cancer gene therapy using adeno-associated virus vectors
Park, K. (2007) · Frontiers in bioscience · doi:10.2741/2872 · Cited by 72 · see citing works
Bosch, M. E., Aldrich, A. L., Fallet, R. W., Odvody, J., Burkovetskaya, M. E., Schuberth, K., Fitzgerald, J. A., Foust, K. D., & Kielian, T. (2016) · Journal of Neuroscience · doi:10.1523/jneurosci.1635-16.2016 · Cited by 70 · see citing works
Liu, W., Zhao, L., Blackman, B. A., Parmar, M. S., Wong, M. Y., Woo, T., Yu, F., Chiuchiolo, M. J., Sondhi, D., Kaminsky, S. M., et al. (2016) · Journal of Neuroscience · doi:10.1523/jneurosci.2016-16.2016 · Cited by 68 · see citing works
Carvalho, L. S., Xiao, R., Wassmer, S. J., Langsdorf, A., Zinn, E., Pacouret, S., Shah, S., Comander, J. I., Kim, L. A., Lim, L., et al. (2018) · Human Gene Therapy · doi:10.1089/hum.2017.154 · Cited by 63 · see citing works
Self-citationTang, W., Szokol, K., Jensen, V., Enger, R., Trivedi, C. A., Hvalby, Ø., Helm, P. J., Looger, L. L., Sprengel, R., & Nagelhus, E. A. (2015) · Journal of Neuroscience · doi:10.1523/jneurosci.3319-14.2015 · Cited by 60 · see citing works
de Leeuw, C. N., Dyka, F. M., Boye, S. L., Laprise, S., Zhou, M., Chou, A., Borretta, L., McInerny, S., Banks, K. G., Portales-Casamar, É., et al. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2013.5 · Cited by 57 · see citing works
Dashkoff, J., Lerner, P., Truong, N., Klickstein, J. A., Fan, Z., Mu, D., Maguire, C. A., Hyman, B. T., & Hudry, E. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.81 · Cited by 56 · see citing works
Self-citationArmbruster, N. S., Lattanzi, A., Jeavons, M., van Wittenberghe, L., Gjata, B., Marais, T., Martin, S., Vignaud, A., Voït, T., Mavilio, F., et al. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.60 · Cited by 54 · see citing works
Shimano, T., Fyk‐Kolodziej, B. E., Mirza, N., Asako, M., Tomoda, K., Bledsoe, S. C., Pan, Z., Molitor, S. C., & Holt, A. G. (2012) · Brain Research · doi:10.1016/j.brainres.2012.10.030 · Cited by 51 · see citing works
Hester, M. E., Foust, K. D., Kaspar, R. W., & Kaspar, B. K. (2009) · Current Gene Therapy · doi:10.2174/156652309789753383 · Cited by 51 · see citing works
Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia
Ajufo, E., & Cuchel, M. (2016) · Current Atherosclerosis Reports · doi:10.1007/s11883-016-0579-0 · Cited by 51 · see citing works
Sargeant, T. J., Wang, S. S., Bradley, J. M., Smith, N. J. C., Raha, A. A., McNair, R., Ziegler, R. J., Cheng, S. H., Cox, T. M., & Cachón-González, M. B. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr364 · Cited by 47 · see citing works
AAV for pain: steps towards clinical translation
Beutler, A. S., & Reinhardt, M. (2009) · Gene Therapy · doi:10.1038/gt.2009.23 · Cited by 44 · see citing works
Wang, L., Xiao, R., Andrés‐Mateos, E., & Vandenberghe, L. H. (2017) · PLoS ONE · doi:10.1371/journal.pone.0182473 · Cited by 41 · see citing works
Self-citationGene Therapy for Psychiatric Disorders
Gelfand, Y. J., & Kaplitt, M. G. (2012) · World Neurosurgery · doi:10.1016/j.wneu.2012.12.028 · Cited by 36 · see citing works
Mak, K. Y., Rajapaksha, I. G., Angus, P., & Herath, C. B. (2017) · Current Gene Therapy · doi:10.2174/1566523217666170314141931 · Cited by 35 · see citing works
Hironaka, K., Yamazaki, Y., Hirai, Y., Yamamoto, M., Miyake, N., Miyake, K., Okada, T., Morita, A., & Shimada, T. (2015) · Scientific Reports · doi:10.1038/srep13104 · Cited by 32 · see citing works
Crommentuijn, M. H. W., Kantar, R. S., Noske, D. P., Vandertop, W. P., Badr, C. E., Wurdinger, T. D., Maguire, C. A., & Tannous, B. A. (2016) · Molecular Therapy — Oncolytics · doi:10.1038/mto.2016.17 · Cited by 30 · see citing works
Self-citationIdentification of the miRNA targetome in hippocampal neurons using RIP-seq
Malmevik, J., Petri, R., Klussendorf, T., Knauff, P., Åkerblom, M., Johansson, J., Soneji, S., & Jakobsson, J. (2015) · Scientific Reports · doi:10.1038/srep12609 · Cited by 30 · see citing works
Davidsson, M., Díaz-Fernández, P., Schwich, O. D., Torroba, M., Wang, G., & Björklund, T. (2016) · Scientific Reports · doi:10.1038/srep37563 · Cited by 27 · see citing works
Tissue-Specific Promoters in the CNS
Kügler, S. (2015) · Methods in molecular biology · doi:10.1007/978-1-4939-3271-9_6 · Cited by 26 · see citing works
Mudannayake, J. M., Mouravlev, A., Fong, D. M., & Young, D. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.75 · Cited by 23 · see citing works
Evaluation of Gene Therapy as an Intervention Strategy to Treat Brain Injury from Stroke
Craig, A. J., & Housley, G. D. (2016) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2016.00034 · Cited by 22 · see citing works
p11 and Gene Therapy for Severe Psychiatric Disorders: A Practical Goal?
Chen, G., Twyman, R. E., & Manji, H. K. (2010) · Science Translational Medicine · doi:10.1126/scitranslmed.3001754 · Cited by 18 · see citing works
Flagged first, then by citations
Cited by
Sorted by citations
Blood-Brain Barrier and Delivery of Protein and Gene Therapeutics to Brain
Pardridge, W. M. (2020) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2019.00373 · Cited by 405 · see citing works
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.02.017 · Cited by 368 · see citing works
Self-citationA Historical Review of Brain Drug Delivery
Pardridge, W. M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14061283 · Cited by 219 · see citing works
Yao, Y., Wang, J., Liu, Y., Qu, Y., Wang, K., Zhang, Y., Chang, Y., Yang, Z., Wan, J., Liu, J., et al. (2022) · Nature Biomedical Engineering · doi:10.1038/s41551-022-00938-7 · Cited by 120 · see citing works
O’Carroll, S. J., Cook, W. H., & Young, D. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.618020 · Cited by 105 · see citing works
Batista, A. R., King, O. D., Reardon, C. P., Davis, C., Shankaracharya, Philip, V. M., Gray‐Edwards, H. L., D, N. A. M., Lutz, C. M., Landers, J. E., et al. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.186 · Cited by 94 · see citing works
Ahmadi, S. E., Soleymani, M., Shahriyary, F., Amirzargar, M. R., Mahya, O., Fattahi, D., & Safa, M. (2023) · Cancer Gene Therapy · doi:10.1038/s41417-023-00597-z · Cited by 95 · see citing works
Biomimetic drug-delivery systems for the management of brain diseases
Chen, Y., Wei, C., Lyu, Y., Chen, H., Jiang, G., & Gao, X. (2019) · Biomaterials Science · doi:10.1039/c9bm01395d · Cited by 86 · see citing works
Astrocytes: Emerging Therapeutic Targets in Neurological Disorders
Valori, C. F., Guidotti, G., Brambilla, L., & Rossi, D. (2019) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2019.04.010 · Cited by 76 · see citing works
Jiang, D., Lee, H., & Pardridge, W. M. (2020) · Scientific Reports · doi:10.1038/s41598-020-70290-w · Cited by 72 · see citing works
Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate
Andrés‐Mateos, E., Landegger, L. D., Unzu, C., Phillips, J., Lin, B. M., Dewyer, N. A., Sanmiguel, J. C., Nicolaou, F., Valero, M. D., Bourdeu, K. I., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-28969-3 · Cited by 64 · see citing works
Self-citationAAV capsid design: A Goldilocks challenge
Zolotukhin, S., & Vandenberghe, L. H. (2022) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2022.01.003 · Cited by 64 · see citing works
Self-citationCRISPR/Cas9 Epigenome Editing Potential for Rare Imprinting Diseases: A Review
Syding, L. A., Nickl, P., Kašpárek, P., & Sedláček, R. (2020) · Cells · doi:10.3390/cells9040993 · Cited by 63 · see citing works
Adeno-Associated Virus Vector for Central Nervous System Gene Therapy
Zhu, D., Schieferecke, A. J., Lopez, P. A., & Schaffer, D. V. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2021.03.010 · Cited by 56 · see citing works
AAV Engineering for Improving Tropism to the Central Nervous System
Ghauri, M. S., & Ou, L. (2023) · Biology · doi:10.3390/biology12020186 · Cited by 61 · see citing works
Brain gene therapy with Trojan horse lipid nanoparticles
Pardridge, W. M. (2023) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2023.02.004 · Cited by 59 · see citing works
Development of New Strategies Using Extracellular Vesicles Loaded with Exogenous Nucleic Acid
Orefice, N. S. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12080705 · Cited by 56 · see citing works
Current and Emerging Strategies for Enhancing Antibody Delivery to the Brain
Bajracharya, R., Caruso, A. C., Vella, L. J., & Nisbet, R. M. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13122014 · Cited by 55 · see citing works
Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations
Hinderer, C. J., Miller, R. C., Dyer, C., Johansson, J., Bell, P. L., Buza, E. L., & Wilson, J. M. (2020) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.51165 · Cited by 51 · see citing works
Humoral immune responses to <scp>AAV</scp> gene therapy in the ocular compartment
Whitehead, M., Osborne, A., Yu‐Wai‐Man, P., & Martin, K. (2021) · Biological Reviews · doi:10.1111/brv.12718 · Cited by 52 · see citing works
Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48 · see citing works
Han, Z., Luo, N., Ma, W., Liu, X., Cai, Y., Kou, J., Wang, J., Li, L., Peng, S., Xu, Z., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-39554-7 · Cited by 46 · see citing works
Brain Delivery of Nanomedicines: Trojan Horse Liposomes for Plasmid DNA Gene Therapy of the Brain
Pardridge, W. M. (2020) · Frontiers in Medical Technology · doi:10.3389/fmedt.2020.602236 · Cited by 47 · see citing works
Routes of administration for adeno-associated viruses carrying gene therapies for brain diseases
Zhou, K., Han, J., Wang, Y., Zhang, Y., & Zhu, C. (2022) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2022.988914 · Cited by 38 · see citing works
Challenges and Opportunities of Targeting Astrocytes to Halt Neurodegenerative Disorders
Valori, C. F., Possenti, A., Brambilla, L., & Rossi, D. (2021) · Cells · doi:10.3390/cells10082019 · Cited by 37 · see citing works
Pardridge, W. M. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114234 · Cited by 37 · see citing works
Genome Editing for CNS Disorders
Duarte, F. T., & Déglon, N. (2020) · Frontiers in Neuroscience · doi:10.3389/fnins.2020.579062 · Cited by 34 · see citing works
Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches
Mijanović, O., Branković, A., Borovjagin, A. V., Butnaru, D. V., Bezrukov, E. A., Sukhanov, R. B., Shpichka, A. I., Timashev, P. S., & Ulasov, I. V. (2020) · Viruses · doi:10.3390/v12040460 · Cited by 32 · see citing works
Fajardo-Serrano, A., Rico, A. J., Roda, E., Honrubia, A., Arrieta, S., Ariznabarreta, G., Chocarro, J., Lorenzo-Ramos, E., Pejenaute, A., Vázquez, A., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22126389 · Cited by 32 · see citing works
Tang, S., Chen, X.-Y., Tong, X.-M., & Zhu, L.-F. (2025) · International Journal of Medical Sciences · doi:10.7150/ijms.112724 · Cited by 23 · see citing works
Pan, X., Sands, S. A., Yue, Y., Zhang, K., LeVine, S. M., & Duan, D. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.008 · Cited by 29 · see citing works
Yoon, S. Y., Hunter, J. E., Chawla, S., Clarke, D. L., Molony, C. M., O’Donnell, P. A., Bagel, J. H., Kumar, M., Poptani, H., Vite, C. H., et al. (2020) · Brain · doi:10.1093/brain/awaa161 · Cited by 28 · see citing works
Ellsworth, J. L., Gingras, J., Smith, L. J., Rubin, H. L., Seabrook, T. A., Patel, K., Zapata, N., Olivieri, K. C., O’Callaghan, M., Chlipala, E. A., et al. (2019) · PLoS ONE · doi:10.1371/journal.pone.0225582 · Cited by 25 · see citing works
AAV Vector-Mediated Antibody Delivery (A-MAD) in the Central Nervous System
Marino, M., & Holt, M. G. (2022) · Frontiers in Neurology · doi:10.3389/fneur.2022.870799 · Cited by 24 · see citing works
Ancestral Library Identifies Conserved Reprogrammable Liver Motif on AAV Capsid
Zinn, E. M., Unzu, C., Schmit, P. F., Turunen, H., Zabaleta, N., Sanmiguel, J. C., Fieldsend, A., Bhatt, U., Diop, C. T., Merkel, E., et al. (2021) · Cell Reports Medicine · doi:10.1016/j.xcrm.2022.100803 · Cited by 25 · see citing works
Self-citationImproved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice
Davidson, C. D., Gibson, A. L., Gu, T., Baxter, L. L., Deverman, B. E., Beadle, K., Incao, A. A., Rodriguez‐Gil, J. L., Fujiwara, H., Jiang, X., et al. (2021) · Life Science Alliance · doi:10.26508/lsa.202101040 · Cited by 23 · see citing works
rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application
Blanc, F., Mondain, M., Bemelmans, A., Affortit, C., Puel, J., & Wang, J. (2020) · Journal of Clinical Medicine · doi:10.3390/jcm9020589 · Cited by 21 · see citing works
Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases
Li, L., Vasan, L., Kartono, B., Clifford, K. P., Attarpour, A., Sharma, R., Mandrozos, M., Kim, A., Zhao, W., Belotserkovsky, A., et al. (2023) · Biomedicines · doi:10.3390/biomedicines11102725 · Cited by 19 · see citing works
A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD
McCallister, T. X., Lim, C. K. W., Singh, M., Zhang, S., Ahsan, N. S., Terpstra, W. M., Xiong, A. Y., C., M. A. Z., Powell, J. E., Drnevich, J. M., et al. (2025) · Nature Communications · doi:10.1038/s41467-024-55548-5 · Cited by 19 · see citing works
Non-viral approaches for gene therapy and therapeutic genome editing across the blood–brain barrier
Xie, R., Wang, Y., Burger, J. C., Li, D., Zhu, M., & Gong, S. (2023) · Med-X · doi:10.1007/s44258-023-00004-0 · Cited by 20 · see citing works
A Single Cisterna Magna Injection of AAV Leads to Binaural Transduction in Mice
Blanc, F., Bemelmans, A., Affortit, C., Joséphine, C., Puel, J., Mondain, M., & Wang, J. (2022) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.783504 · Cited by 17 · see citing works
AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery
Jackson, C. B., Richard, A. S., Ojha, A., Conkright, K. A., Trimarchi, J. M., Bailey, C. C., Alpert, M. D., Kay, M. A., Farzan, M., & Choe, H. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.11.004 · Cited by 16 · see citing works
Adeno-Associated Viruses for Modeling Neurological Diseases in Animals: Achievements and Prospects
Lunev, E. A., Karan, A. A., Egorova, T. V., & Bardina, M. V. (2022) · Biomedicines · doi:10.3390/biomedicines10051140 · Cited by 16 · see citing works
Gene therapy for Lafora disease in the Epm2a mouse model
Zafra‐Puerta, L., Iglesias‐Cabeza, N., Burgos, D. F., Sciaccaluga, M., González‐Fernández, J., Bellingacci, L., Canonichesi, J., Sánchez‐Martín, G., Costa, C., Sánchez, M. P., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.05.032 · Cited by 17 · see citing works
Distributional comparison of different AAV vectors after unilateral cochlear administration
Han, S., Xu, Z., Wang, S., Tang, H., Hu, S., Wang, H., Guan, G., & Shu, Y. (2023) · Gene Therapy · doi:10.1038/s41434-023-00431-z · Cited by 15 · see citing works
Chadman, K. K., Adayev, T., Udayan, A., Ahmed, R., Dai, C., Goodman, J. H., Meeker, H. C., Dolzhanskaya, N., & Velinov, M. T. (2023) · Genes · doi:10.3390/genes14020505 · Cited by 13 · see citing works
Katz, M. G., Hadas, Y., Bailey, R. A., Fazal, S., Vincek, A. S., Madjarova, S. J., Shtraizent, N., Vandenberghe, L. H., & Eliyahu, E. (2021) · Journal of Thoracic and Cardiovascular Surgery · doi:10.1016/j.jtcvs.2021.05.050 · Cited by 11 · see citing works
Self-citationXu, M., Li, J., Xie, J., He, R., Su, Q., Gao, G., & Tai, P. W. L. (2019) · Human Gene Therapy · doi:10.1089/hum.2018.253 · Cited by 11 · see citing works
Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
von Jonquières, G., Rae, C. D., & Housley, G. D. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2021.661857 · Cited by 11 · see citing works
Schwartz, M. K., Likhite, S. B., Vetter, T. A., Baird, M., McGovern, V. L., Sierra-Delgado, J. A., Mendel, T., Burghes, A. H. M., & Meyer, K. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.05.016 · Cited by 10 · see citing works
Sangster, M., Bishop, M., Yao, Y., Feitor, J. F., Shahriar, S., Miller, M. E., Chekuri, A. K., Budnik, B., Bei, F., & Grishchuk, Y. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101269 · Cited by 10 · see citing works
Li, M., Zhong, Y., Zhu, M., Pang, C., Xiao, L. X., Bu, Y., Li, H., Diao, Y., Yang, C. J., & Liu, D. (2024) · The Analyst · doi:10.1039/d4an00404c · Cited by 10 · see citing works
Optimized in vivo base editing restores auditory function in a DFNA15 mouse model
Wang, M., Zhang, Z., Wang, X., Zhang, L., Chen, X., Li, N., Sun, Q., Lu, Y., He, Z., Yang, H., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-63613-w · Cited by 9 · see citing works
SIRT3/GLUT4 signaling activation by metformin protect against cisplatin-induced ototoxicity in vitro
Du, P., Liu, T., Luo, P., Li, H., Tang, W., Zong, S., & Xiao, H. (2023) · Archives of Toxicology · doi:10.1007/s00204-023-03457-9 · Cited by 9 · see citing works
Gene therapy for hereditary hearing loss
Fu, Z., Zhao, L., Guo, Y., & Yang, J. (2024) · Hearing Research · doi:10.1016/j.heares.2024.109151 · Cited by 8 · see citing works
Luchicchi, A., Pattij, T., Viaña, J. N. M., de Kloet, S. F., & Marchant, N. J. (2020) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2020.109004 · Cited by 7 · see citing works
Verdoodt, D., Peeleman, N., Camp, G. V., Rompaey, V. V., & Ponsaerts, P. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2021.728610 · Cited by 8 · see citing works
Advances in designing Adeno-associated viral vectors for development of anti-HBV gene therapeutics
Mnyandu, N., Limani, S. W., Arbuthnot, P. B., & Maepa, M. B. (2021) · Virology Journal · doi:10.1186/s12985-021-01715-9 · Cited by 9 · see citing works
Yu, J.-H., Nam, B.-G., Kim, M., Pyo, S., Seo, J. H., & Cho, S. (2021) · Genes · doi:10.3390/genes12050712 · Cited by 7 · see citing works
Massaro, G., Geard, A. F., Nelvagal, H. R., Gore, K., Clemo, N. K., Waddington, S. N., & Rahim, A. A. (2024) · Human Molecular Genetics · doi:10.1093/hmg/ddae081 · Cited by 5 · see citing works
Pak, A., Wear, D., Tahmasian, N., Min, J. Y., Premraj, D. K., Gibbs, R., Spencer, K. A., Fang, S., Zerbes, T., Krishnan, M. R., et al. (2026) · Frontiers in Neuroscience · doi:10.3389/fnins.2026.1768545 · Cited by 3 · see citing works
Gene Therapy for Fragile X Syndrome, Challenges, and Promises
Velinov, M. T. (2025) · The Journal of Gene Medicine · doi:10.1002/jgm.70049 · Cited by 2 · see citing works
From Gene to Hope: Rett Syndrome and the Rise of Molecular Therapies
Leblay, Y., Félix, M., Roux, J., & Panayotis, N. (2026) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-026-00847-4 · Cited by 2 · see citing works
Cardiac Targeted Adeno-Associated Virus Injection in Rats
Katz, M. G., Hadas, Y., Vincek, A. S., Shtraizent, N., Schadt, E., & Eliyahu, E. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2707-5_10 · Cited by 2 · see citing works
Rybarikova, M., Almacellas‐Barbanoj, A., Schorge, S., & Déglon, N. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.177 · Cited by 2 · see citing works
Hearing impairment: new frontiers of regenerative medicine
Franzè, A., Miceli, M., VECCHIO, V. D., Auletta, G., Laria, C., Malesci, R., & Marciano, E. (2021) · Otorhinolaryngology · doi:10.23736/s2724-6302.21.02381-1 · Cited by 1 · see citing works
Stem Cell Based Exosomes: Are They Effective in Disease or Health?
Kucukguven, M. B., & Çelebi‐Saltik, B. (2021) · Advances in experimental medicine and biology · doi:10.1007/5584_2021_630 · Cited by 1 · see citing works
The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness
Zhou, J. (2025) · American Journal of Stem Cells · doi:10.62347/fesn5062 · Cited by 1 · see citing works
Lau, M., & Hsin, M. K. Y. (2021) · Journal of Thoracic and Cardiovascular Surgery · doi:10.1016/j.jtcvs.2021.06.022 · Cited by 1 · see citing works
Sangster, M., Bishop, M., Yao, Y., Feitor, J. F., Shahriar, S., Miller, M., Chekuri, A. K., Budnik, B., Bei, F., & Grishchuk, Y. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.11.03.565568 · Cited by 1 · see citing works
Gene replacement therapy for Lafora disease in the Epm2a -/- mouse modelPreprint
Zafra‐Puerta, L., Burgos, D. F., Iglesias‐Cabeza, N., González‐Fernández, J., Sánchez‐Martín, G., Sánchez, M. P., & Serratosa, J. M. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.12.14.571636 · Cited by 1 · see citing works
Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders
Casy, W., Ling, Q., Shaffo, F. C., Sinnett, S. E., & Gray, S. J. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-813955-4.00010-6
Commentary: A synthetic adeno-associated viral vector as a means of future precision treatment
Mennander, A. (2021) · Journal of Thoracic and Cardiovascular Surgery · doi:10.1016/j.jtcvs.2021.06.011
Chadman, K. K., Adayev, T., Udayan, A., Ahmed, R., Dai, C., Goodman, J. H., Meeker, H. C., Dolzhanskaya, N., & Velinov, M. T. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.12.22.520936
Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders
Ling, Q., Boitnott, A., Garza, I. T., Casy, W., Shaffo, F. C., Sinnett, S. E., & Gray, S. J. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-19041-4.00007-8
Cochlear Implants: Future Directions
Kara, E., Çögen, T., & Rubinstein, J. T. (2024) · Comprehensive ENT · doi:10.1007/978-3-031-77370-9_28
Enhancing Adeno‐Associated Virus 2 Capsid Engineering Through Co‐Evolutionary Coupling
Laosinwattana, S., Li, Y., Sanchez, B. C., Khan, A., & Dalby, P. A. (2026) · Biotechnology and Bioengineering · doi:10.1002/bit.70349
Khalil, M., Bisignani, M., & Kandler, K. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2025.12.03.692215
The role of gene therapy in neural injury repair
Liu, J., Yuan, A., Tang, Z.-L., & Ning, K. (2026) · Chinese Journal of Traumatology · doi:10.1016/j.cjtee.2025.11.003
Overview of Delivery Methods for Gene Editing
BÜTÜNER, B. D. (2026) · Methods in molecular biology · doi:10.1007/978-1-0716-5547-4_6
Converging Focused Ultrasound and Liquid Biopsy for Glioma Management
Liu, C. J., Woodworth, G. F., & Bettegowda, C. (2026) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2026.08.003
Khalil, M., Bisignani, M., & Kandler, K. (2026) · Frontiers in Genome Editing · doi:10.3389/fgeed.2026.1946992
- Authors
- Hudry, E., Andres-Mateos, E., Lerner, E. P., Volak, A., Cohen, O., Hyman, B. T., Maguire, C. A., & Vandenberghe, L. H.
- Affiliations
- Harvard University (US)
- Broad Institute (US)
- Corresponding author
- Vandenberghe, Luk H.
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2018-09
- Volume
- 10
- Pages
- 197-209
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC6083902
- OpenAlex
- W2884253034
- Cited by
- 84
- References
- 93
- Citations by year
- 2026: 7 · 2025: 6 · 2024: 8 · 2023: 13 · 2022: 13 · 2021: 16 · 2020: 14 · 2019: 7
- FWCI
- 5.66
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- TECNALIA Publications (Fundación TECNALIA Research & Innovation) (submittedVersion, public-domain)
- Funders
- American Brain Tumor Association
- National Institutes of Health
- Cure Alzheimer's Fund
- National Institute on Aging (1K99AG047336-01A1)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- AAV9, blood-brain barrier penetration, intravenous injection, intracerebroventricular administration, intraparenchymal injection, neuronal transduction, cerebellum, striatum, neurological disorders
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
