作者: entrezgene 摘要: This gene encodes a beta polypeptide chain found in adult hemoglobin, which consists of a tetramer of two alpha chains and two beta chains, and which functions in the transport of oxygen to various peripheral tissues. This gene is one of a cluster of beta-hemoglobin ...
Several LV approaches based on gene addition consist in transducing patient HSCs with a lentiviral vector expressing an anti-sickling β-like globin chain such as use of βΑS3 HBB anti-sickling variants. Here, the inventors have improved the design of the LV-AS3 vector to treat SCO ...