NDLI: Survival of the fittest: in vivo selection and stem cell gene therapy

Content Provider	PubMed Central
Author	Neff, Tobias Beard, Brian C. Kiem, Hans-peter
Copyright Year	2006
Abstract	Stem cell gene therapy has long been limited by low gene transfer efficiency to hematopoietic stem cells. Recent years have witnessed clinical success in select diseases such as X-linked severe combined immunodeficiency (SCID) and ADA deficiency. Arguably, the single most important factor responsible for the increased efficacy of these recent protocols is the fact that the genetic correction provided a selective in vivo survival advantage. Since, for most diseases, there will be no selective advantage of gene-corrected cells, there has been a significant effort to arm vectors with a survival advantage. Two-gene vectors can be used to introduce the therapeutic gene and a selectable marker gene. Efficient in vivo selection strategies have been demonstrated in clinically relevant large-animal models. Mutant forms of the DNA repair-enzyme methylguanine methyltransferase in particular have allowed for efficient in vivo selection and have achieved sustained marking with virtually 100% gene-modified cells in large animals, and with clinically acceptable toxicity. Translation of these strategies to the clinical setting is imminent. Here, we review how in vivo selection strategies can be used to make stem cell gene therapy applicable to the treatment of a wider scope of genetic diseases and patients.
Related Links	http://dx.doi.org/10.1182/blood-2005-06-2335
Ending Page	1760
Page Count	10
Starting Page	1751
File Format	PDF
ISSN	00064971
e-ISSN	15280020
Journal	Blood
Issue Number	5
Volume Number	107
Language	English
Publisher	© 2006 by The American Society of Hematology
Publisher Date	2006-03-01
Access Restriction	Open
Rights Holder	© 2006 by The American Society of Hematology
Subject Keyword	Immunology Cell Biology Biochemistry Hematology Research in Higher Education
Content Type	Text
Resource Type	Article
Subject	Cell Biology Hematology Biochemistry Immunology

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