RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

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 · 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

Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., & Wilson, J. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6381-6388.2004 · Cited by 1,070 · see citing works

The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn-/- mice and results in a mouse with spinal muscular atrophy

Monani, U. R. (2000) · Human Molecular Genetics · doi:10.1093/hmg/9.3.333 · Cited by 732 · see citing works

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

Burghes, A. H. M., & Beattie, C. E. (2009) · Nature Reviews Neuroscience · doi:10.1038/nrn2670 · Cited by 723 · see citing works

SMNΔ7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN

Le, T. T., Pham, L. T., Butchbach, M. E. R., Zhang, H. L., Monani, U. R., Coovert, D. D., Gavrilina, T. O., Xing, L., Bassell, G. J., & Burghes, A. H. M. (2005) · Human Molecular Genetics · doi:10.1093/hmg/ddi078 · Cited by 600 · see citing works

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

McCarty, D. M., Fu, H., Monahan, P. E., Toulson, C. E., Naik, P., & Samulski, R. J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302134 · Cited by 547 · see citing works

Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

Maheshri, N., Koerber, J. T., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Biotechnology · doi:10.1038/nbt1182 · Cited by 538 · cited 1× · see citing works

Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue

Duan, D., Sharma, P., Yang, J., Yue, Y., Dudus, L., Zhang, Y., Fisher, K. J., & Engelhardt, J. F. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8568-8577.1998 · Cited by 522 · see citing works

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

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

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

Nakai, H., Yant, S. R., Storm, T. A., Fuess, S., Meuse, L., & Kay, M. A. (2001) · Journal of Virology · doi:10.1128/jvi.75.15.6969-6976.2001 · Cited by 478 · see citing works

Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy

Kariya, S., Park, G.-H., Maeno-Hikichi, Y., Leykekhman, O., Lutz, C. M., Arkovitz, M. S., Landmesser, L. T., & Monani, U. R. (2008) · Human Molecular Genetics · doi:10.1093/hmg/ddn156 · Cited by 445 · see citing works

Impaired Synaptic Vesicle Release and Immaturity of Neuromuscular Junctions in Spinal Muscular Atrophy Mice

Kong, L., Wang, X., Choe, D. W., Polley, M., Burnett, B. G., Bosch‐Marcé, M., Griffin, J. W., Rich, M. M., & Sumner, C. J. (2009) · Journal of Neuroscience · doi:10.1523/jneurosci.4434-08.2009 · Cited by 397 · see citing works

Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy

Murray, L. M., Comley, L. H., Thomson, D., Parkinson, N., Talbot, K., & Gillingwater, T. H. (2007) · Human Molecular Genetics · doi:10.1093/hmg/ddm367 · Cited by 388 · see citing works

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

Avila, A. M., Burnett, B. G., Taye, A. A., Gabanella, F., Knight, M. A., Hartenstein, P., Cizman, Z., Prospero, N. A. D., Pellizzoni, L., Fischbeck, K. H., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci29562 · Cited by 350 · see citing works

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

Asokan, A., Conway, J. C., Phillips, J. L., Li, C., Hegge, J., Sinnott, R., Yadav, S. S., DiPrimio, N., Nam, H. J., Agbandje‐McKenna, M., et al. (2009) · Nature Biotechnology · doi:10.1038/nbt.1599 · Cited by 265 · see citing works

Neurofilament accumulation at the motor endplate and lack of axonal sprouting in a spinal muscular atrophy mouse model

Cifuentes-Díaz, C. (2002) · Human Molecular Genetics · doi:10.1093/hmg/11.12.1439 · Cited by 256 · see citing works

Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

Gavrilina, T. O., McGovern, V. L., Workman, E., Crawford, T. O., Gogliotti, R. G., DiDonato, C. J., Monani, U. R., Morris, G. E., & Burghes, A. H. M. (2008) · Human Molecular Genetics · doi:10.1093/hmg/ddm379 · Cited by 212 · see citing works

Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy

Azzouz, M., Le, T. T., Ralph, G. S., Walmsley, L. E., Monani, U. R., Lee, D. C. P., Wilkes, F. J., Mitrophanous, K. A., Kingsman, S. M., Burghes, A. H. M., et al. (2004) · Journal of Clinical Investigation · doi:10.1172/jci22922 · Cited by 209 · see citing works

Molecular evolution of adeno-associated virus for enhanced glial gene delivery

Koerber, J. T., Klimczak, R., Jang, J.-H., Dalkara, D., Flannery, J. G., & Schaffer, D. V. (2009) · Molecular Therapy · doi:10.1038/mt.2009.184 · Cited by 181 · see citing works

Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates

Kota, J., Handy, C. R., Haidet, A. M., Montgomery, C. L., Eagle, A., Rodino‐Klapac, L. R., Tucker, D., Shilling, C. J., Therlfall, W. R., Walker, C. M., et al. (2009) · Science Translational Medicine · doi:10.1126/scitranslmed.3000112 · Cited by 166 · see citing works

Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition

Narver, H. L., Kong, L., Burnett, B. G., Choe, D. W., Bosch‐Marcé, M., Taye, A. A., Eckhaus, M., & Sumner, C. J. (2008) · Annals of Neurology · doi:10.1002/ana.21449 · Cited by 153 · see citing works

Embryonic motor axon development in the severe SMA mouse

McGovern, V. L., Gavrilina, T. O., Beattie, C. E., & Burghes, A. H. M. (2008) · Human Molecular Genetics · doi:10.1093/hmg/ddn189 · Cited by 150 · see citing works

Abnormal motor phenotype in the SMNΔ7 mouse model of spinal muscular atrophy

Butchbach, M. E. R., Edwards, J. D., & Burghes, A. H. M. (2007) · Neurobiology of Disease · doi:10.1016/j.nbd.2007.04.009 · Cited by 110 · see citing works

Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy

Hastings, M. L., Berniac, J., Liu, Y. H., Abato, P., Jodelka, F. M., Barthel, L., Kumar, S., Dudley, C., Nelson, M. L., Larson, K. C., et al. (2009) · Science Translational Medicine · doi:10.1126/scitranslmed.3000208 · Cited by 87 · see citing works

Decreased Synaptic Activity Shifts the Calcium Dependence of Release at the Mammalian Neuromuscular JunctionIn Vivo

Wang, X., Engisch, K. L., Li, Y., Pinter, M. J., Cope, T. C., & Rich, M. M. (2004) · Journal of Neuroscience · doi:10.1523/jneurosci.2755-04.2004 · Cited by 53 · see citing works

The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9

Saunders, N. R., Ek, C. J., & Dzięgielewska, K. M. (2009) · Nature Biotechnology · doi:10.1038/nbt0909-804 · Cited by 45 · see citing works

Assessment of Liquid Microbead Arrays for the Screening of Newborns for Spinal Muscular Atrophy

Pyatt, R. E., Mihal, D. C., & Prior, T. W. (2007) · Clinical Chemistry · doi:10.1373/clinchem.2007.092312 · Cited by 30 · see citing works

Gene Transfer into the CNS Using Recombinant Adeno-associated Virus: Analysis of Vector DNA Forms Resulting in Sustained Expression

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Tudor reign.

Mackenzie, A. E., & Gendron, N. H. (2001) · Nature Structural Biology · doi:10.1038/82983 · Cited by 10 · see citing works

Adeno-associated virus vector as a platform for gene therapy delivery

Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444 · see citing works

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

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Hinderer, C., Katz, N., Buza, E. L., Dyer, C., Goode, T., Bell, P., Richman, L. K., & Wilson, J. M. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.015 · Cited by 814 · see citing works

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

Hua, Y., Sahashi, K., Rigo, F., Hung, G., Horev, G., Bennett, C. F., & Krainer, A. R. (2011) · Nature · doi:10.1038/nature10485 · Cited by 744 · see citing works

Current clinical applications of in vivo gene therapy with AAVs

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Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelPreprint

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Spinal Muscular Atrophy

Kolb, S. J., & Kissel, J. T. (2015) · Neurologic Clinics · doi:10.1016/j.ncl.2015.07.004 · Cited by 605 · see citing works

Spinal muscular atrophy

D’Amico, A., Mercuri, E. M., Tiziano, F. D., & Bertini, E. (2011) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-6-71 · Cited by 579 · see citing works

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Passini, M. A., Bu, J., Richards, A. M., Kinnecom, C., Sardi, S. P., Stanek, L. M., Hua, Y., Rigo, F., Matson, J. E., Hung, G., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3001777 · Cited by 547 · see citing works

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

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

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Day, J. W., Finkel, R. S., Chiriboga, C. A., Connolly, A. M., Crawford, T. O., Darras, B. T., Iannaccone, S. T., Kuntz, N. L., Peña, L. D. M., Shieh, P. B., et al. (2021) · The Lancet Neurology · doi:10.1016/s1474-4422(21)00001-6 · Cited by 494 · see citing works

Alternative Splicing in the Mammalian Nervous System: Recent Insights into Mechanisms and Functional Roles

Raj, B., & Blencowe, B. J. (2015) · Neuron · doi:10.1016/j.neuron.2015.05.004 · Cited by 475 · see citing works

Spinal muscular atrophy: Diagnosis and management in a new therapeutic era

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Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy

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Spinal muscular atrophy: going beyond the motor neuron

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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

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

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Spinal Muscular Atrophy

Kolb, S. J. (2011) · Archives of Neurology · doi:10.1001/archneurol.2011.74 · Cited by 335 · see citing works

Onasemnogene Abeparvovec: First Global Approval

Hoy, S. M. (2019) · Drugs · doi:10.1007/s40265-019-01162-5 · Cited by 337 · see citing works

Sphingolipid lysosomal storage disorders

Platt, F. M. (2014) · Nature · doi:10.1038/nature13476 · Cited by 320 · see citing works

Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306 · see citing works

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Dominguez, E., Marais, T., Chatauret, N., Benkhelifa-Ziyyat, S., Duque, S., Ravassard, P., Carcenac, R., Astord, S., de Moura, A. P., Voit, T., et al. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddq514 · Cited by 298 · see citing works

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Mendell, J. R., Al-Zaidy, S. A., Lehman, K. J., McColly, M., Lowes, L. P., Alfano, L. N., Reash, N. F., Iammarino, M. A., Church, K. R., Kleyn, A., et al. (2021) · JAMA Neurology · doi:10.1001/jamaneurol.2021.1272 · Cited by 295 · see citing works

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

Zhang, 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

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291 · see citing works

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

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

Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

Rigo, F., Chun, S. J., Norris, D. A., Hung, G., Lee, S., Matson, J., Fey, R. A., Gaus, H., Hua, Y., Grundy, J. S., et al. (2014) · The Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.113.212407 · Cited by 283 · see citing works

Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care

Schorling, D., Pechmann, A., & Kirschner, J. (2019) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-190424 · Cited by 276 · see citing works

Spinal muscular atrophy: a motor neuron disorder or a multi‐organ disease?

Shababi, M., Lorson, C. L., & Rudnik‐Schöneborn, S. (2013) · Journal of Anatomy · doi:10.1111/joa.12083 · Cited by 263 · see citing works

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

Valori, C. F., Ning, K., Wyles, M., Mead, R. J., Grierson, A. J., Shaw, P. J., & Azzouz, M. (2010) · Science Translational Medicine · doi:10.1126/scitranslmed.3000830 · Cited by 285 · see citing works

Overcoming the Blood–Brain Barrier. Challenges and Tricks for CNS Drug Delivery

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Twenty-Five Years of Spinal Muscular Atrophy Research: From Phenotype to Genotype to Therapy, and What Comes Next

Wirth, B., Karakaya, M., Kye, M. J., & Mendoza-Ferreira, N. (2020) · Annual Review of Genomics and Human Genetics · doi:10.1146/annurev-genom-102319-103602 · Cited by 256 · see citing works

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

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Erratum: Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248 · see citing works

Emerging therapies and challenges in spinal muscular atrophy

Farrar, M. A., Park, S. B., Vucic, S., Carey, K. A., Turner, B. J., Gillingwater, T. H., Swoboda, K. J., & Kiernan, M. C. (2016) · Annals of Neurology · doi:10.1002/ana.24864 · Cited by 246 · see citing works

Advances in therapy for spinal muscular atrophy: promises and challenges

Groen, E. J. N., Talbot, K., & Gillingwater, T. H. (2018) · Nature Reviews Neurology · doi:10.1038/nrneurol.2018.4 · Cited by 235 · see citing works

Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy

Al‐Zaidy, S., Pickard, A. S., Kotha, K., Alfano, L. N., Lowes, L., Paul, G., Church, K., Lehman, K., Sproule, D. M., Dabbous, O., et al. (2019) · Pediatric Pulmonology · doi:10.1002/ppul.24203 · Cited by 216 · see citing works

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

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

Spinal Muscular Atrophies

Darras, B. T. (2015) · Pediatric Clinics of North America · doi:10.1016/j.pcl.2015.03.010 · Cited by 213 · see citing works

Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

Bevan, A. K., Hutchinson, K. R., Foust, K. D., Braun, L., McGovern, V. L., Schmelzer, L., Ward, J. G., Petruska, J. C., Lucchesi, P. A., Burghes, A. H. M., et al. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq300 · Cited by 211 · see citing works

Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation

Haery, L., Deverman, B. E., Matho, K. S., Cetin, A. H., Woodard, K., Cepko, C. L., Guerin, K. I., Rego, M. A., Ersing, I., Bächle, S. M., et al. (2019) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2019.00093 · Cited by 207 · see citing works

Spinal muscular atrophy — insights and challenges in the treatment era

Mercuri, E. M., Pera, M. C., Scoto, M., Finkel, R. S., & Francesco Muntoni, A. (2020) · Nature Reviews Neurology · doi:10.1038/s41582-020-00413-4 · Cited by 208 · see citing works

Genetic Correction of Human Induced Pluripotent Stem Cells from Patients with Spinal Muscular Atrophy

Corti, S. P., Nizzardo, M., Simone, C., Falcone, M., Nardini, M., Ronchi, D., Donadoni, C., Salani, S., Riboldi, G. M., Magri, F., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3004108 · Cited by 207 · see citing works

Survival Motor Neuron Protein in Motor Neurons Determines Synaptic Integrity in Spinal Muscular Atrophy

Martinez, T. L., Kong, L., Wang, X., Osborne, M. A., Crowder, M. E., Meerbeke, J. P. V., Xu, X., Davis, C., Wooley, J., Goldhamer, D. J., et al. (2012) · Journal of Neuroscience · doi:10.1523/jneurosci.0204-12.2012 · Cited by 206 · see citing works

Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

Van Alstyne, M., Tattoli, I., Delestrée, N., Recinos, Y., Workman, E., Shihabuddin, L. S., Zhang, C., Mentis, G. Z., & Pellizzoni, L. (2021) · Nature Neuroscience · doi:10.1038/s41593-021-00827-3 · Cited by 188 · see citing works

Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound

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Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy

Ling, K., Gibbs, R. M., Feng, Z., & Ko, C. P. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr453 · Cited by 201 · see citing works

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201 · see citing works

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

Ramos, D. M., d’Ydewalle, C. V. O., Gabbeta, V., Dakka, A., Klein, S., Norris, D. A., Matson, J. E., Taylor, S. J., Zaworski, P. G., Prior, T. W., et al. (2019) · Journal of Clinical Investigation · doi:10.1172/jci124120 · Cited by 190 · 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

Gene therapy for ALS: A review

Amado, D. A., & Davidson, B. L. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.04.008 · Cited by 192 · see citing works

Spinal Muscular Atrophy: A Clinical and Research Update

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