Article

Expression and processing of mature human frataxin after gene therapy in mice

2023-12-28

Abstract excerpt

Friedreich’s ataxia is a degenerative and progressive multisystem disorder caused by mutations in the highly conserved frataxin (FXN) gene that results in FXN protein deficiency and mitochondrial dysfunction. While gene therapy approaches are promising, consistent induction of therapeutic FXN protein expression that is sub-toxic has proven challenging, and numerous therapeutic approaches are being tested in animal...

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Literature Corpus work: 52da10c5-c27b-5b2b-a84e-320e5357920a
DOI: 10.21203/rs.3.rs-3788652/v1

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