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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Veröffentlicht in:Mol Ther Methods Clin Dev
Hauptverfasser: Kiem, Hans-Peter, Arumugam, Paritha I, Burtner, Christopher R, Fox, Catherine F, Beard, Brian C, Dexheimer, Phillip, Adair, Jennifer E, Malik, Punam
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2014
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Article
Online Zugang: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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