Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm
نویسندگان
چکیده
منابع مشابه
Gene Therapy for β - Thalassemia
Gene transfer for β-thalassemia requires gene transfer into hematopoietic stem cells using integrating vectors that direct regulated expression of β globin at therapeutic levels. Among integrating vectors, oncoretroviral vectors carrying the human βglobin gene and portions of the locus control region (LCR) have suffered from problems of vector instability, low titers and variable expression. In...
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ژورنال
عنوان ژورنال: HemaSphere
سال: 2021
ISSN: 2572-9241
DOI: 10.1097/hs9.0000000000000555