Genomic safe harbors permit high -globin transgene expression in thalassemia induced pluripotent stem cells

If transgenes are to be introduced into the genome for cell therapies, the integration events should permit high transgene expression without altering the expression of endogenous genes. Papapetrou et al. propose five criteria to define such 'safe harbors' in the human genome and demonstra...

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Bibliographic Details
Published in:Nature biotechnology 2011-01, Vol.29 (1), p.73
Main Authors: Papapetrou, Eirini P, Lee, Gabsang, Malani, Nirav, Setty, Manu, Riviere, Isabelle, Tirunagari, Laxmi M S, Kadota, Kyuichi
Format: Article
Language:English
Subjects:
Analysis
Bioinformatics
Computational biology
Criteria
Gene expression
Genes
Genetic engineering
Genetic research
Genetically modified organisms
Genomes
Genomics
Health aspects
Human genome
MicroRNA
Neomycin
Stem cells
Suicide
Thalassemia
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Summary:If transgenes are to be introduced into the genome for cell therapies, the integration events should permit high transgene expression without altering the expression of endogenous genes. Papapetrou et al. propose five criteria to define such 'safe harbors' in the human genome and demonstrate high A-globin expression from a safe-harbor site in erythroid-lineage cells derived from induced pluripotent stem (iPS) cells.
ISSN:1087-0156
1546-1696
DOI:10.1038/nbt.1717