CL20-4i-EF1α-hγc-OPT transduced CD34+ hematopoietic stem cells

CL20-4i-EF1α-hγc-OPT transduced CD34+ hematopoietic stem cells is an investigational ex vivo gene therapy developed by the National Institute of Allergy and Infectious Diseases (NIAID) for the treatment of X-linked severe combined immunodeficiency (SCID-X1). The therapy involves the collection of autologous CD34+ hematopoietic stem cells, which are then transduced with a third-generation, self-inactivating lentiviral vector (CL20-i4-EF1α-hγc-OPT). This vector utilizes the eukaryotic elongation factor 1-alpha (EF1α) promoter to drive the expression of a codon-optimized human common gamma chain (γc) cDNA, compensating for mutations in the IL2RG gene. The vector design includes a 400-bp chicken β-globin insulator fragment to enhance safety and transgene expression. Following transduction, the modified cells are reinfused into the patient, typically after low-dose busulfan conditioning, to restore functional immune system development.

Other names

Lentiviral Gene Transfer for X-Linked SCIDNIAID SCID-X1 Gene Therapyhγc-OPT transduced CD34+ HSCs