Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes

MACDONALD, M. (1993) · Cell · doi:10.1016/0092-8674(93)90585-e · Cited by 8,366

Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

Rosen, D. R., Siddique, T., Patterson, D., Figlewicz, D. A., Sapp, P., Hentati, A., Donaldson, D., Goto, J., O'Regan, J. P., Deng, H.-X., et al. (1993) · Nature · doi:10.1038/362059a0 · Cited by 7,062

Ubiquitinated TDP-43 in Frontotemporal Lobar Degeneration and Amyotrophic Lateral Sclerosis

Neumann, M., Sampathu, D. M., Kwong, L. K., Truax, A. C., Micsenyi, M. C., Chou, T. T., Bruce, J., Schuck, T., Grossman, M., Clark, C. M., et al. (2006) · Science · doi:10.1126/science.1134108 · Cited by 6,576

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

DeJesus-Hernandez, M., Mackenzie, I. R., Boeve, B. F., Boxer, A. L., Baker, M., Rutherford, N. J., Nicholson, A. M., Finch, N. A., Flynn, H., Adamson, J., et al. (2011) · Neuron · doi:10.1016/j.neuron.2011.09.011 · Cited by 4,936 · cited 3×

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

Renton, A. E., Majounie, E., Waite, A., Simón-Sánchez, J., Rollinson, S., Gibbs, J. R., Schymick, J. C., Laaksovirta, H., van Swieten, J. C., Myllykangas, L., et al. (2011) · Neuron · doi:10.1016/j.neuron.2011.09.010 · Cited by 4,409

Identification and characterization of a spinal muscular atrophy-determining gene

Lefebvre, S., Bürglen, L., Reboullet, S., Clermont, O., Burlet, P., Viollet, L., Bénichou, B., Cruaud, C., Millasseau, P., Zeviani, M., et al. (1995) · Cell · doi:10.1016/0092-8674(95)90460-3 · Cited by 3,995

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group.

Shah, M. A., Datson, N. A., Srinidhi, L., Stanton, V. P., MacDonald, M. E., Allard, M. W., Youngman, S., Am, F., Mott, R., Draths, K., et al. (1993) · Cell · Cited by 3,055

Mechanisms of gene silencing by double-stranded RNA

Meister, G., & Tuschl, T. (2004) · Nature · doi:10.1038/nature02873 · Cited by 2,598

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Finkel, R. S., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Saito, K., Servais, L., Tizzano, E., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1702752 · Cited by 2,337

Amyotrophic lateral sclerosis

Hardiman, O. M., Al‐Chalabi, A., Chiò, A., Corr, E. M., Logroscino, G., Robberecht, W., Shaw, P. J., Simmons, Z., & van den Berg, L. H. (2017) · Nature Reviews Disease Primers · doi:10.1038/nrdp.2017.71 · Cited by 2,237

Pathological phase transitions in ALS-FTD impair dynamic RNA–protein granules

Nedelsky, N. B., & Taylor, J. P. (2021) · RNA · doi:10.1261/rna.079001.121 · Cited by 31

Knockdown of polypyrimidine tract binding protein facilitates motor function recovery after spinal cord injury

Yang, R., Chai, R., Pan, J.-Y., Pan, J., Xia, P.-H., Wang, Y.-K., Chen, Y., Li, Y., Wu, J., & Chen, G. (2022) · Neural Regeneration Research · doi:10.4103/1673-5374.346463 · Cited by 30

Nucleic acid therapies for CNS diseases: Pathophysiology, targets, barriers, and delivery strategies

Padmakumar, S., D’souza, A. A., Parayath, N. N., Bleier, B. S., & Amiji, M. M. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.10.018 · Cited by 30

Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide

Yeoh, Y. Q., Amin, A., Cuic, B., Tomas, D., Turner, B. J., & Shabanpoor, F. (2024) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2024.116737 · Cited by 29

Molecular Insight Into the Therapeutic Potential of Long Non-coding RNA-Associated Competing Endogenous RNA Axes in Alzheimer’s Disease: A Systematic Scoping Review

Sabaie, H., Amirinejad, N., Asadi, M. R., Jalaiei, A., Daneshmandpour, Y., Rezaei, O., Taheri, M. N., & Rezazadeh, M. (2021) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2021.742242 · Cited by 29

Biosafety assessment of delivery systems for clinical nucleic acid therapeutics

Li, Z., Zhang, L., Jiang, K., Zhang, Y., Liu, Y., Hu, G., & Song, J. (2022) · Biosafety and Health · doi:10.1016/j.bsheal.2022.03.003 · Cited by 29

The nexus of long noncoding RNAs, splicing factors, alternative splicing and their modulations

Malakar, P., Shukla, S. K., Mondal, M., Kar, R. K., & Siddiqui, J. A. (2023) · RNA Biology · doi:10.1080/15476286.2023.2286099 · Cited by 28

Conventional protein kinase C in the brain: repurposing cancer drugs for neurodegenerative treatment?

Lordén, G., & Newton, A. C. (2021) · Neuronal Signaling · doi:10.1042/ns20210036 · Cited by 27

mTORC2 mediates structural plasticity in distal nociceptive endings that contributes to pain hypersensitivity following inflammation

Wong, C., Barkai, O., Wang, F., Pérez, C. T., Lev, S., Cai, W., Tansley, S. N., Yousefpour, N., Hooshmandi, M., Lister, K. C., et al. (2022) · Journal of Clinical Investigation · doi:10.1172/jci152635 · Cited by 28

Liver‐directed drugs for t ransthyretin‐mediated amyloidosis

Brannagan, T. H., Berk, J. L., Gillmore, J. D., Maurer, M. S., Waddington‐Cruz, M., Fontana, M., Masri, A., Obici, L. P., Brambatti, M., Baker, B. F., et al. (2022) · Journal of the Peripheral Nervous System · doi:10.1111/jns.12519 · Cited by 31

Real-life Wrist Movement Patterns Capture Motor Impairment in Individuals with Ataxia-Telangiectasia

Gupta, A. S., Luddy, A. C., Khan, N. C., Reiling, S., & Thornton, J. K. (2022) · The Cerebellum · doi:10.1007/s12311-022-01385-5 · Cited by 26

Safety and Efficacy of Nusinersen and Risdiplam for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials

Qiao, Y., Chi, Y., Gu, J., & Ma, Y. (2023) · Brain Sciences · doi:10.3390/brainsci13101419 · Cited by 27

Antisense Oligonucleotide In Vitro Protein Binding Determination in Plasma, Brain, and Cerebral Spinal Fluid Using Hybridization LC-MS/MS

Guimaraes, G. J., Yuan, L., & Li, P. S. (2021) · Drug Metabolism and Disposition · doi:10.1124/dmd.121.000751 · Cited by 25

Nuclear Magnetic Resonance Spectra and AMBER OL3 and ROC-RNA Simulations of UCUCGU Reveal Force Field Strengths and Weaknesses for Single-Stranded RNA

Zhao, J., Kennedy, S. D., & Turner, D. H. (2022) · Journal of Chemical Theory and Computation · doi:10.1021/acs.jctc.1c00643 · Cited by 26

Non-coding RNA-directed therapeutics in lung cancer: Delivery technologies and clinical applications

Abdellatif, A. A. H., Scagnetti, G., Younis, M. A., Bouazzaoui, A., Tawfeek, H. M., Aldosari, B. N., Almurshedi, A. S., Alsharidah, M. S., Rugaie, O. A., Davies, M. P. A., et al. (2023) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2023.113466 · Cited by 25

Cutting-edge advances in modeling the blood–brain barrier and tools for its reversible permeabilization for enhanced drug delivery into the brain

Sharma, A., Fernandes, D. C., Reis, R. L., Gołubczyk, D., Neumann, S., Łukomska, B., Janowski, M., Kortylewski, M., Walczak, P., Oliveira, J. M., et al. (2023) · Cell & Bioscience · doi:10.1186/s13578-023-01079-3 · Cited by 26

Antisense oligonucleotides enhance SLC20A2 expression and suppress brain calcification in a humanized mouse model

Zhao, M., Cheng, X., Chen, L., Zeng, Y., Lin, K.-J., Li, Y., Zheng, Z.-H., Xue-jing, H., Zuo, D., Guo, X.-X., et al. (2024) · Neuron · doi:10.1016/j.neuron.2024.07.013 · Cited by 26

Current potential therapeutics of amyotrophic lateral sclerosis

Lu, L., Deng, Y., & Xu, R. (2024) · Frontiers in Neurology · doi:10.3389/fneur.2024.1402962 · Cited by 26

RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington’s disease models

Lin, Y., Li, C., Chen, Y., Gao, J., Li, J., Huang, C., Liu, Z., Wang, W., Zheng, X., Song, X., et al. (2025) · Molecular Neurodegeneration · doi:10.1186/s13024-024-00794-w · Cited by 26

Mechanistic research and therapeutic prospects of alternative splicing in neurodegenerative diseases

Ran, X., Wang, M., Huang, J., Kuang, N., Tian, P., Wu, J., Feng, F., Luo, Y., & Huang, N. (2026) · Ageing Research Reviews · doi:10.1016/j.arr.2026.103133 · Cited by 24

Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment

O’Callaghan, B. L., Hofstra, B., Handler, H. P., Kordasiewicz, H. B., Cole, T. A., Duvick, L. A., Friedrich, J., Rainwater, O., Yang, P., Benneyworth, M. A., et al. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.07.030 · Cited by 24

Binding of phosphorothioate oligonucleotides with RNase H1 can cause conformational changes in the protein and alter the interactions of RNase H1 with other proteins

Zhang, L., Vickers, T. A., Sun, H., Liang, X., & Crooke, S. T. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab078 · Cited by 24

Therapeutic Development for CGG Repeat Expansion-Associated Neurodegeneration

Xu, K., Li, Y., Allen, E. G., & Jin, P. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2021.655568 · Cited by 25

Inflammatory Non-CpG Antisense Oligonucleotides Are Signaling Through TLR9 in Human Burkitt Lymphoma B Bjab Cells

Pollak, A. J., Cauntay, P., Machemer, T., Paz, S., Damle, S. S., Henry, S. P., & Burel, S. A. (2022) · Nucleic Acid Therapeutics · doi:10.1089/nat.2022.0034 · Cited by 24

Noncoding RNAs and Midbrain DA Neurons: Novel Molecular Mechanisms and Therapeutic Targets in Health and Disease

Pascale, E., Divisato, G., Palladino, R., Auriemma, M., Ngalya, E. F., & Caiazzo, M. (2020) · Biomolecules · doi:10.3390/biom10091269 · Cited by 24

Genome-scale exon perturbation screens uncover exons critical for cell fitness

Xiao, M., Damodaran, A. P., Kumari, B., Dickson, E., Xing, K., On, T. A., Parab, N., King, H. E., Perez, A. R., Guiblet, W. M., et al. (2024) · Molecular Cell · doi:10.1016/j.molcel.2024.05.024 · Cited by 23

Understanding In Vivo Fate of Nucleic Acid and Gene Medicines for the Rational Design of Drugs

Fumoto, S., Yamamoto, T., Okami, K., Maemura, Y., Terada, C., Yamayoshi, A., & Nishida, K. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13020159 · Cited by 23

Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains

Guo, L., Mann, J. R., Mauna, J. C., Copley, K. E., Wang, H. H., Rubien, J. D., Bergmann, C. A., Carey, J., Merjane, J., Ngo, M., et al. (2026) · Molecular Cell · doi:10.1016/j.molcel.2025.12.009 · Cited by 24

Self-assembly of DNA nanogels with endogenous microRNA toehold self-regulating switches for targeted gene regulation therapy

Yan, J., Zou, H., Zhou, W., Yuan, X., Li, Z., Ma, X., Liu, C., Wang, Y., Rosenholm, J. M., Cui, W., et al. (2022) · Biomaterials Science · doi:10.1039/d2bm00640e · Cited by 22

Tau kinetics in Alzheimer's disease

Hier, D. B., Azizi, S., Thimgan, M. S., & Wunsch, D. C. (2022) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2022.1055170 · Cited by 22

RNAi in cell nuclei: potential for a new layer of biological regulation and a new strategy for therapeutic discovery

Johnson, K. C., & Corey, D. R. (2023) · RNA · doi:10.1261/rna.079500.122 · Cited by 21

Site-directed A → I RNA editing as a therapeutic tool: moving beyond genetic mutations

Quiroz, J. F. D., Siskel, L. D., & Rosenthal, J. J. C. (2023) · RNA · doi:10.1261/rna.079518.122 · Cited by 21

CRISPR in Neurodegenerative Diseases Treatment: An Alternative Approach to Current Therapies

Akbar, A., Haider, R., Agnello, L., Noor, B., Maqsood, N., Atif, F., Ali, W., Ciaccio, M., & Tariq, H. (2025) · Genes · doi:10.3390/genes16080850 · Cited by 21

Self‐Assembled Antibody‐Oligonucleotide Conjugates for Targeted Delivery of Complementary Antisense Oligonucleotides

Zhou, L., Bi, J., Chang, S., Bai, Z., Yu, J., Wang, R., Li, Z., Zhang, X., Chou, J. J., & Pan, L. (2024) · Angewandte Chemie International Edition · doi:10.1002/anie.202415272 · Cited by 20

Alternative splicing at neuroligin site A regulates glycan interaction and synaptogenic activity

Oku, S., Feng, H., Connor, S. A., Toledo, A., Zhang, P., Zhang, Y., Thoumine, O. R., Zhang, C., & Craig, A. M. (2020) · eLife · doi:10.7554/elife.58668 · Cited by 21

Oligonucleotide-based therapeutics for neurodegenerative disorders: Focus on antisense oligonucleotides

Mansour, H. M., & El-Khatib, A. S. (2025) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2025.177529 · Cited by 20

Structural Context of a Critical Exon of Spinal Muscular Atrophy Gene

Singh, N. N., O’Leary, C. A., Eich, T., Moss, W. N., & Singh, R. N. (2022) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2022.928581 · Cited by 19

The development of peptide- and oligonucleotide-based drugs to prevent the formation of abnormal tau in tauopathies

Lozupone, M., Dibello, V., Sardone, R., Castellana, F., Zupo, R., Lampignano, L., Bortone, I., Stallone, R., Altamura, M., Bellomo, A., et al. (2023) · Expert Opinion on Drug Discovery · doi:10.1080/17460441.2023.2200245 · Cited by 21

A sequential binding mechanism for 5′ splice site recognition and modulation for the human U1 snRNP

White, D. S., Dunyak, B. M., Vaillancourt, F. H., & Hoskins, A. A. (2024) · Nature Communications · doi:10.1038/s41467-024-53124-5 · Cited by 19

Targeting tau in Alzheimer’s and beyond: Insights into pathology and therapeutic strategies

Singh, S., Khan, S., Shahid, M., Sardar, M., Hassan, M. I., & Islam, A. (2024) · Ageing Research Reviews · doi:10.1016/j.arr.2024.102639 · Cited by 19

Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model

Berciano, M. T., Puente-Bedia, A., Medina-Samamé, A., Rodríguez-Rey, J. C., Calderó, J., Lafarga, M., & Tapia, O. (2020) · Scientific Reports · doi:10.1038/s41598-020-67569-3 · Cited by 19

RNA-Targeting Techniques: A Comparative Analysis of Modern Approaches for RNA Manipulation in Cancer Research and Therapeutics

Boti, M. A., Diamantopoulos, M. A., & Scorilas, A. (2025) · Genes · doi:10.3390/genes16101168 · Cited by 20

Progress towards drug discovery for Friedreich’s Ataxia: Identifying synthetic oligonucleotides that more potently activate expression of human frataxin protein

Shen, X., Wong, J., Prakash, T. P., Rigo, F., Li, Y., Напиерала, М., & Corey, D. R. (2020) · Bioorganic & Medicinal Chemistry · doi:10.1016/j.bmc.2020.115472 · Cited by 19

Antisense Transcription across Nucleotide Repeat Expansions in Neurodegenerative and Neuromuscular Diseases: Progress and Mysteries

Castro, A. F., Loureiro, J. R., Bessa, J., & Silveira, I. (2020) · Genes · doi:10.3390/genes11121418 · Cited by 18

High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome

Ottesen, E. W., Luo, D., Singh, N. N., & Singh, R. N. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22168378 · Cited by 18

The brain targeted delivery of programmed cell death 4 specific siRNA protects mice from CRS-induced depressive behavior

Jia, Y., Zhuang, X., Zhang, Y., Zhao, M., Chen, N., Li, W., Zhu, F., Guo, C., Li, Y., Wang, Q., et al. (2021) · Cell Death & Disease · doi:10.1038/s41419-021-04361-9 · Cited by 17

Scientific Review of the Proarrhythmic Risks of Oligonucleotide Therapeutics: Are Dedicated ICH S7B / E14 Studies Needed for Low‐Risk Modalities?

Qu, Y., Henderson, K. A., Harper, T. A., & Vargas, H. M. (2024) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.3204 · Cited by 17

Neuronal Intranuclear Inclusion Disease with NOTCH2NLC GGC Repeat Expansion: A Systematic Review and Challenges of Phenotypic Characterization

Zeng, T. S., Chen, Y., Huang, H., Li, S., Huang, J., Xie, H., Lin, S., Chen, S., Chen, G., & Yang, D. (2024) · Aging and Disease · doi:10.14336/ad.2024.0131-1 · Cited by 17

l-Type amino acid transporter 1-targeting nanoparticles for antisense oligonucleotide delivery to the CNS

Lim, Y. N., Ryu, I. S., Jung, Y.-J., Helmlinger, G., Kim, I., Park, H. W., Kang, H., Lee, J., Lee, H. J., Lee, K. S., et al. (2024) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2024.102340 · Cited by 17

Unraveling the multifaceted insights into amyotrophic lateral sclerosis: Genetic underpinnings, pathogenesis, and therapeutic horizons

Sharma, R., Khan, Z., Mehan, S., Gupta, G. D., & Narula, A. S. (2024) · Mutation Research/Reviews in Mutation Research · doi:10.1016/j.mrrev.2024.108518 · Cited by 18

Protein Kinase CK2 and Its Potential Role as a Therapeutic Target in Huntington’s Disease

White, A., McGlone, A., & Gómez‐Pastor, R. (2022) · Biomedicines · doi:10.3390/biomedicines10081979 · Cited by 17

Radiolabeling of functional oligonucleotides for molecular imaging

Liu, D., Xia, Q., Ding, D., & Tan, W. (2022) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2022.986412 · Cited by 19

Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.

Metovic, J., Li, Y., Gong, Y., & Eichler, F. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00443 · Cited by 16

Advances in Nucleic Acid Research: Exploring the Potential of Oligonucleotides for Therapeutic Applications and Biological Studies

Moccia, M., Pascucci, B., Saviano, M., Cerasa, M. T., Terzidis, M. A., Chatgilialoglu, C., & Masi, A. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms25010146 · Cited by 16

Antisense targeting of decoy exons can reduce intron retention and increase protein expression in human erythroblasts

Parra, M., Zhang, W., Vu, J. T., DeWitt, M. A., & Conboy, J. G. (2020) · RNA · doi:10.1261/rna.075028.120 · Cited by 16

Advancing precision medicines for ocular disorders: Diagnostic genomics to tailored therapies

Panikker, P., Roy, S., Ghosh, A., Poornachandra, B., & Ghosh, A. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2022.906482 · Cited by 17

More is Needed to Complement the Available Therapies of Spinal Muscular Atrophy

Singh, R. N. (2019) · Future Medicinal Chemistry · doi:10.4155/fmc-2019-0239 · Cited by 16

Overview of Methods for Large-Scale RNA Synthesis

Ryczek, M., Mateja-Pluta, M., Błaszczyk, L., & Kiliszek, A. (2022) · Applied Sciences · doi:10.3390/app12031543 · Cited by 15

Coaxial Synthesis of PEI-Based Nanocarriers of Encapsulated RNA-Therapeutics to Specifically Target Muscle Cells

de la Hoz, R., Diban, N., Berciano, M. T., Emeterio, C. S., Urtiaga, A. M., Lafarga, M., Rodríguez-Rey, J. C., & Tapia, O. (2022) · Biomolecules · doi:10.3390/biom12081012 · Cited by 15

A mechanistic pharmacokinetic model for intrathecal administration of antisense oligonucleotides

Linninger, A. A., Barua, D., Hang, Y., Iadevaia, S., & Vakilynejad, M. (2023) · Frontiers in Physiology · doi:10.3389/fphys.2023.1130925 · Cited by 15

RNA‐Based Therapies for Neurodegenerative Diseases Targeting Pathogenic Proteins

Rabbani, S. A., El‐Tanani, M., Sharma, S., El‐Tanani, Y., Kumar, R., Saini, M., Yadav, M., Khan, M. A., & Parvez, S. (2025) · European Journal of Neuroscience · doi:10.1111/ejn.70110 · Cited by 15

Human Induced Pluripotent Stem Cell-Based Modelling of Spinocerebellar Ataxias

Hommersom, M. P., Buijsen, R. A. M., van Roon‐Mom, W., van de Warrenburg, B. P. C., & van Bokhoven, H. (2021) · Stem Cell Reviews and Reports · doi:10.1007/s12015-021-10184-0 · Cited by 15

New Insights into lncRNAs in Aβ Cascade Hypothesis of Alzheimer’s Disease

Hao, Y., Xie, B., Fu, X., Xu, R., & Yang, Y. F. (2022) · Biomolecules · doi:10.3390/biom12121802 · Cited by 16

NET formation-mediated in situ protein delivery to the inflamed central nervous system

Wu, Y., Park, J.-W., Le, Q., Byun, J., Choi, J., Xu, E., Lee, J., & Oh, Y. (2024) · Nature Communications · doi:10.1038/s41467-024-54817-7 · Cited by 14

A Novel Gene Therapy for Neurodegenerative Lafora Disease Via Epm2A -Loaded Dlindma Lipoplexes

Vemana, H. P., Saraswat, A. L., Bhutkar, S. P., Patel, K. R., & Dukhande, V. V. (2021) · Nanomedicine · doi:10.2217/nnm-2020-0477 · Cited by 14

Brain Repair by Cell Replacement via In Situ Neuronal Reprogramming

Qian, H., & Fu, X. (2021) · Annual Review of Genetics · doi:10.1146/annurev-genet-071719-023616 · Cited by 14

Microflow liquid chromatography – multi-emitter nanoelectrospray mass spectrometry of oligonucleotides

Guimaraes, G. J., Leach, F. E., & Bartlett, M. G. (2023) · Journal of Chromatography A · doi:10.1016/j.chroma.2023.463976 · Cited by 14

Development of a novel severe mouse model of spinal muscular atrophy with respiratory distress type 1: FVB-nmd

Shababi, M., Smith, C. E., Garro-Kacher, M. O., Rawi, Z. A., Villalón, E., Davis, D. J., Bryda, E. C., & Lorson, C. L. (2019) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2019.10.032 · Cited by 14

A survey of transcripts generated by spinal muscular atrophy genes

Singh, N. N., Ottesen, E. W., & Singh, R. N. (2020) · Biochimica et Biophysica Acta (BBA) - Gene Regulatory Mechanisms · doi:10.1016/j.bbagrm.2020.194562 · Cited by 15

RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System

Ertural, B., Çiçek, B. N., & Kurnaz, I. A. (2025) · Biomolecules & Therapeutics · doi:10.4062/biomolther.2025.022 · Cited by 13

Aptamer Reduces Aggregation of Mutant Huntingtin and Rescues Proteostasis Network in Non-Neuronal and Neuronal Cells

Jain, S., & Roy, I. (2023) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.3c00226 · Cited by 13

Harnessing miRNAs: A Novel Approach to Diagnosis and Treatment of Tuberculosis

Doghish, A. S., Abulsoud, A. I., Nassar, Y. A., Nasr, S. M., Mohammed, O. A., Abdel‐Reheim, M. A., Rizk, N. I., Lutfy, R. H., Mageed, S. S. A., Ismail, M. S., et al. (2025) · Journal of Biochemical and Molecular Toxicology · doi:10.1002/jbt.70119 · Cited by 13

Targeting PTB for Glia-to-Neuron Reprogramming In Vitro and In Vivo for Therapeutic Development in Neurological Diseases

Contardo, M., Gioia, R. D., Gagliardi, D., Comi, G. P., Ottoboni, L., Nizzardo, M., & Corti, S. P. (2022) · Biomedicines · doi:10.3390/biomedicines10020399 · Cited by 14

Upregulation of the ESCRT pathway and multivesicular bodies accelerates degradation of proteins associated with neurodegeneration

Benyair, R., Giridharan, S. S. P., Rivero-Ríos, P., Hasegawa, J., Bristow, E., Eskelinen, E., Shmueli, M. D., Fishbain-Yoskovitz, V., Merbl, Y., Sharkey, L. M., et al. (2023) · Autophagy Reports · doi:10.1080/27694127.2023.2166722 · Cited by 13

Global alternative splicing landscape of skeletal muscle atrophy induced by hindlimb unloading

Sun, J., Yang, H., Yang, X., Chen, X., Xu, H. Z., Shen, Y., Ding, F., Gu, X., Zhu, J., & Sun, H. (2021) · Annals of Translational Medicine · doi:10.21037/atm-20-5388 · Cited by 13

Targeting Poison Exons to Treat Developmental and Epileptic Encephalopathy

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