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Title of Journal: Int J Hematol

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Abbravation: International Journal of Hematology

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Springer Japan

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DOI

10.1002/malq.19630091802

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ISSN

1865-3774

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Hematopoietic Stem Cell Gene Therapy

Authors: David W Emery Tamon Nishino Ken Murata Michalis Fragkos George Stamatoyannopoulos
Publish Date: 2002/04/01
Volume: 75, Issue: 3, Pages: 228-236
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Abstract

Gene therapy applications that target hematopoietic stem cells HSCs offer great potential for the treatment of hematologic disease Despite this promise clinical success has been limited by poor rates of gene transfer poor engraftment of modified cells and poor levels of gene expression We describe here the basic approach used for HSC gene therapy briefly review some of the seminal clinical trials in the field and describe several recent advances directed toward overcoming these limitations

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