Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing

Disorders involving β-globin gene mutations, primarily β-thalassemia and sickle cell disease, represent a major target for hematopoietic stem/progenitor cell (HSPC) gene therapy. This includes CRISPR/Cas9-mediated genome editing approaches in adult CD34(+) cells aimed toward the reactivation of feta...

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Vydáno v:Blood
Hlavní autoři: Li, Chang, Psatha, Nikoletta, Sova, Pavel, Gil, Sucheol, Wang, Hongjie, Kim, Jiho, Kulkarni, Chandana, Valensisi, Cristina, Hawkins, R. David, Stamatoyannopoulos, George, Lieber, André
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2018
Témata:
Gene Therapy
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6024639/
https://ncbi.nlm.nih.gov/pubmed/29789357
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2018-03-838540
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