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Evaluation of cardiolipin nanodisks as lipid replacement therapy for Barth syndrome
Barth syndrome (BTHS) is a mitochondrial disorder characterized by cardiomyopathy and skeletal muscle weakness. Disease results from mutations in the tafazzin (TAZ) gene, encoding a phospholipid transacylase. Defective tafazzin activity results in an aberrant cardiolipin (CL) profile. The feasibilit...
Autores principales: | Ikon, Nikita, Hsu, Fong-Fu, Shearer, Jennifer, Forte, Trudy M., Ryan, Robert O. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Editorial Department of Journal of Biomedical Research
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5895564/ https://www.ncbi.nlm.nih.gov/pubmed/29336355 http://dx.doi.org/10.7555/JBR.32.20170094 |
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