pSRS11.EFS.IL2RG

pSRS11.EFS.IL2RG is an investigational self-inactivating (SIN) gammaretroviral gene therapy vector developed for the treatment of X-linked severe combined immunodeficiency (X-SCID), also known as SCID-X1. The vector is designed to deliver a functional copy of the human *IL2RG* cDNA, which encodes the interleukin-2 receptor subunit gamma (the common gamma chain, or γc). Unlike first-generation gene therapies that used intact viral long-terminal repeats (LTRs) and were associated with insertional mutagenesis leading to T-cell leukemia, the pSRS11.EFS.IL2RG vector features a SIN design where the enhancer/promoter elements of the LTRs are deleted. Gene expression is instead driven by an internal, mammalian elongation factor 1α short (EFS) promoter, which is intended to reduce the risk of transactivating neighboring oncogenes. The therapeutic process involves the ex vivo transduction of autologous CD34+ hematopoietic stem cells with the vector, followed by re-infusion into the patient to restore lymphocyte development and immune function.

Other names

SCID-X1 SIN gammaretroviral vectorSCID-X-1 SIN gammaretroviral vectorSCID-X 1 SIN gammaretroviral vectorSelf-inactivating gamma-retroviral vector expressing IL2RG