Safety, β-Sarcoglycan Expression, and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4

Autor: Jerry R. Mendell, E. R. Pozsgai, Linda Lowes, S. Neuhaus, M. A. Iammarino, A. S. Meadows, Kelly J. Lehman, Danielle A. Griffin, N. F. Reash, E. Koenig, A. Mueller-York, Sarah Lewis, L. R. Rodino-Klapac, X. Li, Kathleen Church
Rok vydání: 2021
Předmět:
Zdroj: Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics.
ISSN: 1439-1899
DOI: 10.1055/s-0041-1739648
Databáze: OpenAIRE