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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Bibliografiske detaljer
Udgivet i: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
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2014
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Article
Online adgang: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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