Official websites use .gov
A
.gov website belongs to an official
government organization in the United States.
Secure .gov websites use HTTPS
A lock (
) or https:// means you've safely
connected to the .gov website. Share sensitive
information only on official, secure websites.
Overview of human β-globin locus and ex vivo gene therapy for the treatment of SCD and TDT. A) Schematic representation of human β-globin locus and LCR; B) Erythroid-specific expression of β-like globin transgenes using lentiviral vectors; C) Schematic representation of lentiviral gene addition therapy and HBB mutation-independent therapeutic approaches for the treatment of SCD and TDT.
