AAV-gene therapy in galactosemia patient fibroblasts

What this episode covers

Dr Megan Brophy and Dr Bob Bell join the podcast to talk about their recent work looking at AAV gene therapy in fibroblasts from patients with classic galactosemia. We discuss new insights into disease physiology and consider the challenges of scaling gene therapy towards in vivo model. AAV-mediated expression of galactose-1-phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts Megan L. Brophy, et al https://doi.org/10.1002/jimd.12468

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Frequently Asked Questions

How long is this episode of JIMD Podcasts?

This episode is 12 minutes long.

When was this JIMD Podcasts episode published?

This episode was published on February 25, 2022.

What is this episode about?

Dr Megan Brophy and Dr Bob Bell join the podcast to talk about their recent work looking at AAV gene therapy in fibroblasts from patients with classic galactosemia. We discuss new insights into disease physiology and consider the challenges of...

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