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Gene Therapy

Researchers' Work from University of North Carolina Focuses on Gene Therapy

Published in Gene Therapy Weekly, December 22nd, 2011

2011 DEC 22 -- Researchers detail in "Gene therapy in skeletal muscle mediated by adeno-associated virus vectors," new data in Gene Therapy. According to the authors of recent research from Chapel Hill, North Carolina, "Adeno-associated virus (AAV) is the most promising gene delivery vehicle for muscle-directed gene therapy. AAV's natural tropism to muscle cells, long-term persistent transgene expression, multiple serotypes, as well as its minimal immune response have made AAV vectors well suited for muscle-directed gene therapy."

"AAV vector-mediated gene delivery to augment muscle structural proteins, such as dystrophin and sarcoglycans, offers great hope...

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