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Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

The beta-thalassemias are inherited anemias caused by mutations that severely reduce or abolish expression of the beta-globin gene. Like sickle cell disease, a related beta-globin gene disorder, they are ideal candidates for performing a genetic correction in patient hematopoietic stem cells (HSCs)....

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Hum Gene Ther
Egile Nagusiak: Mansilla-Soto, Jorge, Riviere, Isabelle, Boulad, Farid, Sadelain, Michel
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2016
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC4994056/
https://ncbi.nlm.nih.gov/pubmed/27021486
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.037
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