AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome

Barth syndrome (BTHS) is a rare mitochondrial disease that affects heart and skeletal muscle and has no curative treatment. It is caused by recessive mutations in the X-linked gene TAZ, which encodes tafazzin. To develop a clinically relevant gene therapy to restore tafazzin function and treat BTHS,...

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Опубликовано в: :Hum Gene Ther
Главные авторы: Suzuki-Hatano, Silveli, Saha, Madhurima, Rizzo, Skylar A., Witko, Rachael L., Gosiker, Bennett J., Ramanathan, Manashwi, Soustek, Meghan S., Jones, Michael D., Kang, Peter B., Byrne, Barry J., Cade, W. Todd, Pacak, Christina A.
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc., publishers 2019
Предметы:
Research Articles
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6383582/
https://ncbi.nlm.nih.gov/pubmed/30070157
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.020
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