Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

Gene therapy of severe hemoglobinopathies will require high-level expression of a transferred globin gene in erythroid cells. Distant regulatory elements flanking the beta-globin gene cluster, the locus control region, are needed for appropriate expression. We have explored the use of a human parvov...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Walsh, C E, Liu, J M, Xiao, X, Young, N S, Nienhuis, A W, Samulski, R J
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 1992
Gaiak:
Research Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC49685/
https://ncbi.nlm.nih.gov/pubmed/1323131
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