Gene Targeting

Study Demonstrates Feasibility of Correcting Mutant Genes in Hematopoietic Stem Cells

Published in Gene Therapy Weekly, December 28th, 2000

The first report of using homologous recombination to repair genetic mutations in hematopoietic stem cells suggests promise for this strategy.

Researchers at the University of North Carolina, Chapel Hill, and at Niigata University, Japan, conducted their gene targeting experiment in hematopoietic progenitor cells in culture, noting that these cells are "more abundant and more mature and so less pluripotent" than hematopoietic stem cells (HSCs). They reported their findings in the the Proceedings of the National Academy of Sciences ("Gene correction in hematopoietic progenitor cells by homologous recombination," PNAS, December 5,...

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Source: Gene Therapy Weekly (2000-12-28)

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