Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach for gene therapy of β-thalassemia and sickle cell disease. Using the CRISPR/Cas9 system, we genetically modified human hematopoietic stem and progenitor cells (HSPCs) to mimic the large rearrangements in th...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Lattanzi, Annalisa, Meneghini, Vasco, Pavani, Giulia, Amor, Fatima, Ramadier, Sophie, Felix, Tristan, Antoniani, Chiara, Masson, Cecile, Alibeu, Olivier, Lee, Ciaran, Porteus, Matthew H., Bao, Gang, Amendola, Mario, Mavilio, Fulvio, Miccio, Annarita
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6318785/
https://ncbi.nlm.nih.gov/pubmed/30424953
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.10.008
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