Gene therapy in a mouse model of MSUD - JIMD Podcasts

What this episode covers

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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Frequently Asked Questions

How long is this episode of JIMD Podcasts?

This episode is 13 minutes long.

When was this JIMD Podcasts episode published?

This episode was published on June 9, 2023.

What is this episode about?

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...

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