Morales, Pablo R

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Foust, K. D., Wang, X., McGovern, V. L., Braun, L., Bevan, A. K., Haidet, A. M., Le, T. T., Morales, P. R., Rich, M. M., Burghes, A. H. M., et al. (2010) · Nature Biotechnology

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

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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

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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

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Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

Cain, J. T., Likhite, S., White, K. A., Timm, D. J., Davis, S. S., Johnson, T. B., Dennys-Rivers, C. N., Rinaldi, F., Motti, D., Corcoran, S., et al. (2019) · Molecular Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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