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A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

Reactivation of fetal hemoglobin (HbF) is a promising approach for the treatment of β-hemoglobinopathies and the targeting of genes involved in HbF regulation is under intensive investigation. Here, we established a nonhuman primate (NHP) transplantation model to evaluate hematopoietic stem cell (HS...

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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: Humbert, Olivier, Peterson, Christopher W., Norgaard, Zachary K., Radtke, Stefan, Kiem, Hans-Peter
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2017
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5738324/
https://ncbi.nlm.nih.gov/pubmed/29276718
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.11.005
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