Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in β-thalassemic mice

Globin gene transfer in autologous hematopoietic stem cells is a promising therapeutic option for subjects with β-thalassemia major. In this approach, high level, erythroid-specific globin transgene expression should correct ineffective erythropoiesis and hemolytic anemia following the delivery of o...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Lisowski, Leszek, Sadelain, Michel
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Hematology 2007
Gaiak:
Gene Therapy
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC2234778/
https://ncbi.nlm.nih.gov/pubmed/17921347
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2007-08-108647
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