autologous CD34+ cells transduced with pSRS11.EFS.IL2RG.pre

autologous CD34+ cells transduced with pSRS11.EFS.IL2RG.pre is an investigational ex vivo gene therapy developed for the treatment of X-linked severe combined immunodeficiency (SCID-X1). The therapy utilizes a self-inactivating (SIN) gammaretroviral vector, pSRS11.EFS.IL2RG.pre, which incorporates the human elongation factor 1 alpha (EFS) promoter to drive the expression of the IL2RG gene. This gene encodes the common gamma chain (CD132), a critical subunit of receptors for interleukins including IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21, which are essential for the development and maturation of T cells and natural killer (NK) cells. By transducing a patient's own CD34+ hematopoietic stem cells with this vector, the therapy aims to restore immune competence. The SIN vector design and the use of an internal EFS promoter were specifically engineered to minimize the risk of insertional mutagenesis and leukemogenesis that was observed with first-generation retroviral vectors used in early SCID-X1 trials. Following re-infusion, these modified stem cells engraft in the bone marrow and differentiate into a functional immune system.

Other names

SIN-gammaretroviral vector-transduced CD34+ cellsCD34+ cells transduced with pSRS11.EFS.IL2RG.pre