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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Detalles Bibliográficos
Autores principales: Kiem, Hans-Peter, Arumugam, Paritha I, Burtner, Christopher R, Fox, Catherine F, Beard, Brian C, Dexheimer, Phillip, Adair, Jennifer E, Malik, Punam
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4448740/
https://www.ncbi.nlm.nih.gov/pubmed/26052523
http://dx.doi.org/10.1038/mtm.2014.55

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4448740/
https://www.ncbi.nlm.nih.gov/pubmed/26052523
http://dx.doi.org/10.1038/mtm.2014.55

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