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Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells

Induced pluripotent stem (iPS) cell technology holds vast promises for a cure to the hemoglobinopathies. Constructs and methods to safely insert therapeutic genes to correct the genetic defect need to be developed. Site-specific insertion is a very attractive method for gene therapy because the risk...

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Detalhes bibliográficos
Main Authors: Chang, Chan-Jung, Bouhassira, Eric E.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3543982/
https://ncbi.nlm.nih.gov/pubmed/23002118
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-03-420703
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