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Autor:
Markus Grompe, Gregory N. Clarke, Carl Pelz, Ronald W. McClard, Amy E. Hanlon Newell, Karsten Wursthorn, Nicole K. Paulk, Milton J. Finegold, John F. Witte, Susan B. Olson, Cary O. Harding, Annelise Haft, Raymond L Bateman
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 20(10)
Genetic fumarylacetoacetate hydrolase (Fah) deficiency is unique in that healthy gene-corrected hepatocytes have a strong growth advantage and can repopulate the diseased liver. Unfortunately, similar positive selection of gene-corrected cells is abs