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pSRS11.EFS.IL2RG.pre* retroviral vector
- Development stage
- Unknown
- Lead developer
- University College London
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
pSRS11.EFS.IL2RG.pre* is a next-generation, self-inactivating (SIN) gammaretroviral gene therapy vector developed for the treatment of X-linked severe combined immunodeficiency (SCID-X1). It was engineered to address the safety concerns of earlier first-generation vectors by deleting the enhancer/promoter region of the viral long terminal repeat (LTR), thereby reducing the risk of insertional mutagenesis and oncogenesis. The vector utilizes a human elongation factor 1α short (EFS) promoter to drive the expression of a codon-optimized human IL2RG complementary DNA (cDNA), which encodes the common gamma chain (γc) protein. The therapy is administered ex vivo, where autologous CD34+ hematopoietic stem cells are transduced with the vector and subsequently re-infused into the patient to restore immune function by enabling the development of T, B, and NK cells.
- Other names
- SIN-gammaretroviral vector encoding IL2RGEFS-IL2RG retroviral vectorEFS-IL-2RG retroviral vectorEFS-IL 2RG retroviral vector
02
Targets
IL2RG (Interleukin-2 receptor gamma subunit)
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