Mendell, Jerry R.

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

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 2,381

Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

Haidet-Phillips, A. M., Hester, M. E., Miranda, C. J., Meyer, K., Braun, L., Frakes, A., Song, S., Likhite, S., Murtha, M. J., Foust, K. D., et al. (2011) · Nature Biotechnology

Amyotrophic Lateral Sclerosis ResearchAlzheimer's disease research and treatments

Cited by 782

Current clinical applications of in vivo gene therapy with AAVs

Mendell, J. R., Al-Zaidy, S. A., Rodino-Klapac, L. R., Goodspeed, K., Gray, S. J., Kay, C. N., Boye, S. L., Boye, S. E., George, L. A., Salabarria, S., et al. (2021) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 743

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

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 494

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

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

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 337

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

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 295

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

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 286

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

Neurogenetic and Muscular Disorders ResearchLysosomal Storage Disorders Research

Cited by 216

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

Al-Zaidy, S. A., Kolb, S. J., Lowes, L., Alfano, L. N., Shell, R., Church, K. R., Nagendran, S., Sproule, D. M., Feltner, D. E., Wells, C., et al. (2019) · Journal of Neuromuscular Diseases

Neurogenetic and Muscular Disorders ResearchLysosomal Storage Disorders Research

Cited by 185

Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy

Lowes, L. P., Alfano, L. N., Arnold, W. D., Shell, R., Prior, T. W., McColly, M., Lehman, K. J., Church, K., Sproule, D. M., Nagendran, S., et al. (2019) · Pediatric Neurology

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 179

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

Mendell, J. R., Connolly, A. M., Lehman, K. J., Griffin, D. A., Khan, S. Z., Dharia, S. D., Quintana-Gallardo, L., & Rodino-Klapac, L. R. (2022) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 124

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Day, J. W., Mendell, J. R., Mercuri, E., Finkel, R. S., Strauss, K. A., Kleyn, A., Tauscher-Wisniewski, S., Tukov, F. F., Reyna, S. P., & Chand, D. H. (2021) · Drug Safety

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 119

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Day, J. W., Finkel, R. S., Mercuri, E., Swoboda, K. J., Menier, M., van Olden, R., Tauscher-Wisniewski, S., & Mendell, J. R. (2021) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 48