ALAS2 lentiviral vector

The ALAS2 lentiviral vector (also known as X-ALAS2-LV) is an erythroid-specific gene therapy candidate developed for the treatment of X-linked sideroblastic anemia (XLSA). XLSA is a monogenic disorder caused by mutations in the ALAS2 gene, which encodes the erythroid-specific 5-aminolevulinate synthase 2, the rate-limiting enzyme in heme biosynthesis. The vector is designed to deliver a functional human ALAS2 gene specifically to erythroid cells, thereby restoring heme production and correcting the ineffective erythropoiesis and iron overload characteristic of the disease. Preclinical studies in mouse models have demonstrated that the vector can rescue lethal anemia, normalize hemoglobin levels, and reduce systemic iron accumulation. The program is currently in the preclinical research stage, led by investigators at the Children's Hospital of Philadelphia.

Other names

ALAS2-expressing lentiviral vectorALAS-2-expressing lentiviral vectorALAS 2-expressing lentiviral vector