AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome

Barth syndrome (BTHS) is a rare mitochondrial disease that causes severe cardiomyopathy and has no disease-modifying therapy. It is caused by recessive mutations in the gene tafazzin (TAZ), which encodes tafazzin—an acyltransferase that remodels the inner mitochondrial membrane lipid cardiolipin. To...

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Detalles Bibliográficos
Autores principales: Suzuki-Hatano, Silveli, Saha, Madhurima, Soustek, Meghan S., Kang, Peter B., Byrne, Barry J., Cade, W. Todd, Pacak, Christina A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6369239/
https://www.ncbi.nlm.nih.gov/pubmed/30788385
http://dx.doi.org/10.1016/j.omtm.2019.01.007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6369239/
https://www.ncbi.nlm.nih.gov/pubmed/30788385
http://dx.doi.org/10.1016/j.omtm.2019.01.007

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