Restoration of beta-Globin Expression with Optimally Designed Lentiviral Vector for beta-Thalassemia Treatment in Chinese Patients

Ouyang, WJ; Dong, GY; Zhao, WH; Li, J; Zhou, ZH; Yang, GH; Liu, RR; Li, Y; Zhang, QX; Du, X; Sun, HX; Gu, Y; Lai, YR; Liu, SX; Liu, C

Gu, Y; Liu, C (corresponding author), BGI Shenzhen, Shenzhen 518083, Peoples R China.; Lai, YR (corresponding author), Guangxi Med Univ, Affiliated Hosp 1, Dept Hematol, Nanning 530021, Peoples R China.; Liu, SX (corresponding author), Shenzhen Childrens Hosp, Dept Hematol & Oncol, Shenzhen 518038, Peoples R China.

HUMAN GENE THERAPY, 2021; 32 (9-10): 481

Abstract

beta-Thalassemia is one of the most prevalent genetic diseases worldwide. The current treatment for beta-thalassemia is allogeneic hematopoietic stem ......

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