Gene therapy in a mouse model of MSUD
Jun 09, 2023•14 min
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Episode description
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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