Published in Gene Therapy Weekly, March 10th, 1997
Because of its large size and protein synthesizing capabilities, the liver is an important target for gene therapy. Diseases that may benefit from liver-directed gene therapy include various hematological or metabolic disorders.
Dwight D. Koeberl and colleagues from several institutions including the Fred Hutchinson Cancer Research Center, Washington, collaborated on the development of AAV vectors with potential for hepatic gene transfer ("Persistent Expression of Human Clotting Factor IX...
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