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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Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: Kiem, Hans-Peter, Arumugam, Paritha I, Burtner, Christopher R, Fox, Catherine F, Beard, Brian C, Dexheimer, Phillip, Adair, Jennifer E, Malik, Punam
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2014
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Article
Länkar: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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