Al-Zaidy, Samiah

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

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

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial

Mendell, J. R., Sahenk, Z., Lehman, K., Nease, C., Lowes, L. P., Miller, N. F., Iammarino, M. A., Alfano, L. N., Nicholl, A., Al-Zaidy, S., et al. (2020) · JAMA Neurology

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 405

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

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