Selective Expansion of Transduced Cells for Hematopoietic Stem Cell Gene Therapy View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2002-11-01

AUTHORS

Akihiro Kume, Yutaka Hanazono, Hiroaki Mizukami, Takashi Okada, Keiya Ozawaa

ABSTRACT

Although gene transfer into hematopoietic stem cells holds a considerable therapeutic potential, clinical trials targeting this cell compartment have achieved limited success. Poor transduction efficiency with gene transfer vectors used in human studies has hindered delivering therapeutic genes to clinically relevant numbers of target cells. One way to overcome the lowefficiency problem is by selecting or expanding the number of genetically modified cells to a suprathreshold level to achieve clinical efficacy. This approach may be further classified into 2 categories: one is to transfer a drug resistance gene and eliminate unmodified cells with cytotoxic drugs, and the other is to confer a direct growth advantage on target cells.This review aims at an overview of recent advances involving these strategies, with some details of “selective amplifier genes,” a novel system that we have developed for specific expansion of genetically modified hematopoietic cells.Int J Hematol. 2002;76:299-304. More... »

PAGES

299-304

Identifiers

URI

http://scigraph.springernature.com/pub.10.1007/bf02982687

DOI

http://dx.doi.org/10.1007/bf02982687

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1025528598

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/12463591


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