Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies

Safely achieving long-term engraftment of genetically modified hematopoietic stem cells (HSCs) that maintain therapeutic transgene expression is the benchmark for successful application of gene therapy for hemoglobinopathies. We used the pigtailed macaque HSC transplantation model to ascertain the l...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Kiem, Hans-Peter, Arumugam, Paritha I, Burtner, Christopher R, Fox, Catherine F, Beard, Brian C, Dexheimer, Phillip, Adair, Jennifer E, Malik, Punam
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4448740/
https://ncbi.nlm.nih.gov/pubmed/26052523
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2014.55
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