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Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration

Ex vivo gene correction of hematopoietic stem and progenitor cells (HSPCs) has emerged as a promising therapeutic approach for treatment of inherited human blood disorders. Use of engineered nucleases to target therapeutic transgenes to their endogenous genetic loci addresses many of the limitations...

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Podrobná bibliografie
Vydáno v:Mol Ther
Hlavní autoři: Bloomer, Hanan, Smith, Richard H., Hakami, Waleed, Larochelle, Andre
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2021
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8058434/
https://ncbi.nlm.nih.gov/pubmed/33309880
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.12.010
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