Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma

The potential of gene therapy as a curative treatment for monogenetic disorders has been clearly demonstrated in a series of recent Phase I/II clinical trials. Among primary immunodeficiencies, gene transfer into hematopoietic stem (HSC)/progenitor cells has resulted in the long-term correction of i...

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Bibliographic Details
Main Authors: Grez, Manuel, Reichenbach, Janine, Schwäble, Joachim, Seger, Reinhard, Dinauer, Mary C, Thrasher, Adrian J
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2011
Subjects:
Review
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3017455/
https://ncbi.nlm.nih.gov/pubmed/21045810
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.232
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