Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

Maple syrup urine disease (MSUD) is a rare inborn error of metabolism, which is currently treated with life-long low-protein diet that can be challenging to maintain. Here the authors develop an AAV8-directed gene therapy providing sustainable disease rescue in a mouse model of MSUD.

Bibliographic Details
Main Authors: Clément Pontoizeau, Marcelo Simon-Sola, Clovis Gaborit, Vincent Nguyen, Irina Rotaru, Nolan Tual, Pasqualina Colella, Muriel Girard, Maria-Grazia Biferi, Jean-Baptiste Arnoux, Agnès Rötig, Chris Ottolenghi, Pascale de Lonlay, Federico Mingozzi, Marina Cavazzana, Manuel Schiff
Format: Article
Language:English
Published: Nature Portfolio 2022-06-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-30880-w

Internet

https://doi.org/10.1038/s41467-022-30880-w

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