EPISODE · Jun 9, 2023 · 13 MIN
Gene therapy in a mouse model of MSUD
from JIMD Podcasts · host Journal of Inherited Metabolic Disease
Dr Manuel Schiff and Dr Clément Pontoizeau join us for the first JIMD Podcast on Maple Syrup Urine Disease, hi-lighting their recent successes treating a Bckdhb knock-out mouse model using gene therapy. Successful treatment of severe MSUD in Bckdhb−/− mice with neonatal AAV gene therapy Clément Pontoizeau, et al https://doi.org/10.1002/jimd.12604
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Gene therapy in a mouse model of MSUD
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