EF1αS-ADA lentiviral vector gene-modified autologous CD34+ cells

EF1αS-ADA lentiviral vector gene-modified autologous CD34+ cells is an ex vivo autologous gene therapy developed for the treatment of adenosine deaminase deficiency causing severe combined immunodeficiency (ADA-SCID). The therapy involves harvesting autologous CD34+ hematopoietic stem and progenitor cells from the patient's bone marrow or peripheral blood and transducing them ex vivo with a self-inactivating HIV-1-based lentiviral vector. This vector carries the human ADA cDNA under the control of a shortened elongation factor 1 alpha (EF1αS or EFS) promoter, which is engineered to maintain high-level transcription in hematopoietic cells while reducing the potential for trans-activation of neighboring genes. Once re-infused into the patient, these modified cells engraft in the bone marrow and differentiate into various immune cell lineages, providing a continuous source of the functional ADA enzyme to restore metabolic and immune function. The product was primarily developed by Great Ormond Street Hospital (GOSH) and University College London (UCL), with Orchard Therapeutics later acquiring the rights to the commercial version known as OTL-101.

Other names

EFS-ADAEFS-ADA LVautologous CD34+ cells transduced with EFS-ADA lentiviral vectorEF1αS-ADA lentiviral vector gene-modified autologous CD34+ cells