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4D using directed evolution to optimize AAV vectors for gene therapy
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4D: AAV (r)evolution
Monday, July 14, 2014
Molecular Therapeutics LLC is using libraries of
capsid-mutated adeno-associated virus vectors as a starting point for directed
evolution of new vectors to overcome challenges in delivering gene therapy.
have emerged as a preferred strategy for gene therapy because they can
effectively deliver therapeutic amounts of protein and are generally considered
safe. The only approved gene therapy, uniQure
N.V.'s Glybera alipogene tiparvovec, uses an AAV vector to
deliver the missing gene in lipoprotein lipase deficiency (LPLD).
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