EPrints - Publications Repository

Raghav, Rahul and Awata, Junya and Martin, Gregory L. and Strathdee, Douglas and Blanton, Robert M. and Chin, Michael T. (2024) A cell penetrating peptide enhances tafazzin gene therapy in a mouse model of Barth Syndrome. International Journal of Molecular Sciences, 25 (24): 13560. ISSN 1661-6596

AI Summary:

Researchers have developed a gene therapy for Barth Syndrome BTHS, an early onset and lethal X-linked disorder. The therapy uses a cell-penetrating peptide to enhance the delivery of the TAFAZZIN gene.

AI Topics:

The following topics are generate through an AI service. Clicking on them will take you to a search result page with related items.

Abstract

Barth Syndrome (BTHS) is an early onset, lethal X-linked disorder caused by a mutation in tafazzin (TAFAZZIN), a mitochondrial acyltransferase that remodels monolysocardiolipin (MLCL) to mature cardiolipin (CL) and is essential for normal mitochondrial, cardiac, and skeletal muscle function. Current gene therapies in preclinical development require high levels of transduction. We tested whether TAFAZZIN gene therapy could be enhanced with the addition of a cell-penetrating peptide, penetratin (Antp). We found that TAFAZZIN-Antp was more effective than TAFAZZIN at preventing the development of pathological cardiac hypertrophy and heart failure. These findings indicate that a cell-penetrating peptide enhances gene therapy for BTHS.

People