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International Journal of Cell Cloning, Vol 8, 80-96, Copyright © 1990 by AlphaMed Press

REVIEWS

Targeted gene modification for gene therapy of stem cells

SS Boggs
Department of Radiation Oncology, University of Pittsburgh School of Medicine, Pennsylvania.

Ideally, gene therapy would correct the specific gene defect without adding potentially harmful extraneous DNA sequences. Such correction can be obtained with homologous recombination between input DNA sequences and identical (homologous) sequences in the genomic target gene. The development of techniques for obtaining virtually pure populations of hematopoietic stem cells should permit the use of the highly efficient nuclear microinjection methods for transfer of DNA. These techniques combined with new highly sensitive methods for detecting cells with the specified genetic modification of nonexpressed genes would make homologous recombination-mediated gene therapy feasible for hematopoietic stem cells. These advances are reviewed with particular emphasis on approaches to targeted gene modification of hematopoietic stem cells and speculation on directions for future research.


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W. Anderson
Human gene therapy
Science, May 8, 1992; 256(5058): 808 - 813.
[Abstract] [PDF]


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