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Gene Therapy

Reports from University of North Carolina Highlight Recent Research in Gene Therapy

Published in Gene Therapy Weekly, January 27th, 2011

2011 JAN 27 -- Fresh data on Gene Therapy are presented in the report 'Directed evolution of adeno-associated virus (AAV) as vector for muscle gene therapy.' According to a study from the United States, "Adeno-associated virus (AAV) is emerging as a vector of choice for muscle gene therapy because of its effective and stable transduction in striated muscles. AAV naturally evolve into multiple serotypes with diverse capsid gene sequences that are apparently the determinants of their tissue tropism and infectivity."

"Certain AAV serotypes show robust gene transfer upon direct intramuscular injection, while others are effective in crossing the endothelial...

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