Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice

X-linked severe combined immunodeficiency (X-SCID) has been successfully treated by hematopoietic stem cell (HSC) transduction with retroviral vectors expressing the interleukin-2 receptor subunit gamma gene (IL2RG), but several patients developed malignancies due to vector integration near cellular...

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Udgivet i:Mol Ther
Main Authors: Hiramoto, Takafumi, Li, Li B., Funk, Sarah E., Hirata, Roli K., Russell, David W.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5993949/
https://ncbi.nlm.nih.gov/pubmed/29606506
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.02.028
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