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CL20-4i-EF1α-hγc-OPT transduced CD34+ hematopoietic stem cells
- Development stage
- Unknown
- Lead developer
- National Institute of Allergy and Infectious Diseases
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
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
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