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Gene Therapy

Data from Washington University Advance Knowledge in Gene Therapy

Published in Gene Therapy Weekly, December 22nd, 2011

2011 DEC 22 -- Investigators discuss in "AAV-mediated liver-directed gene therapy" new findings in Gene Therapy. "The liver is directly or indirectly involved in many essential processes and is affected by numerous inherited diseases. Therefore, many inherited diseases could be effectively treated by targeting the liver using gene transfer approaches," scientists writing in the journal Methods In Molecular Biology report.

"The challenges associated with liver-directed gene therapy are efficient targeting of hepatocytes, stability of the vector genome, and persistent high level expression. Many of these obstacles can be overcome with adeno-associated...

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