Lehman, Kelly J.

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

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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

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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

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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

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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

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