Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials

Segura, EER; Ayoub, PG; Hart, KL; Kohn, DB

Kohn, DB (通讯作者)，Univ Calif Los Angeles, David Geffen Sch Med, Dept Microbiol Immunol & Mol Genet, Los Angeles, CA 90095 USA.;Kohn, DB (通讯作者)，Univ Calif Los Angeles, David Geffen Sch Med, Dept Pediat Hematol Oncol, Los Angeles, CA 90095 USA.;Kohn, DB (通讯作者)，Univ Calif Los Angeles, Eli & Edythe Broad Ctr Stem Cell Res & Regenerat M, Los Angeles, CA 90095 USA.

VIRUSES-BASEL, 2023; 15 (3):

Abstract

Investigations to understand the function and control of the globin genes have led to some of the most exciting molecular discoveries and biomedical b......

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