Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice

Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice

The leukodystrophy Canavan disease is a fatal white matter disorder caused by loss-of-function mutations of the aspartoacylase-encoding ASPA gene. There are no effective treatments available and experimental gene therapy trials have failed to provide sufficient amelioration from Canavan disease symp...

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Bibliographic Details
Main Authors: Dominik Fröhlich, Elizabeth Kalotay, Georg von Jonquieres, Andre Bongers, Brendan Lee, Alexandra K. Suchowerska, Gary D. Housley, Matthias Klugmann
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-12-01
Series:Frontiers in Molecular Neuroscience
Subjects:
Canavan disease
leukodystrophy
gene therapy
adeno-associated virus
aspartoacylase
ASPA
Online Access:https://www.frontiersin.org/articles/10.3389/fnmol.2022.1061257/full

Internet

https://www.frontiersin.org/articles/10.3389/fnmol.2022.1061257/full

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