Lentiviral Vector Development for X-SCID
To address the efficacy and safety of gene therapy for X-linked severe combined immunodeficiency (X-SCID), Creative Biolabs has developed an LV carrying a codon-optimized human IL2RG cDNA, driven by a short promoter. This vector can be potentially used for gene therapy for X-SCID. We are fully competent and dedicated to serving as your one-stop-shop for LV development for X-SCID.
The Background of X-SCID
X-SCID is caused by loss-of-function mutations in the γc gene (IL2RG), which encodes the common γ-chain (γc) subunit for the IL2, IL4, IL7, IL9, IL15, and IL21 receptors and is required for signal transduction with these cytokines. Human X-SCID is characterized by lack of T, natural killer (NK) cells, and nonfunctional B cells and is fatal early in life from progressive infections if left untreated.
Gene Therapy of X-SCID
Allogeneic stem cell transplantation is the current standard of care for X-SCID and can be curative, particularly when a matched sibling donor is available. However, most patients lack a matched sibling donor. Most experts agree that better treatment is desirable for X-SCID patients who lack a matched sibling donor. Gene therapy is one of several approaches and is being developed as an alternative to haploidentical allogeneic transplantation. Transplantation of hematopoietic stem/progenitor cells (HSPCs) genetically corrected with early murine leukemia retrovirus (MLV)-derived gammaretroviral vectors have shown restoration of T cell immunity in patients, but it results in vector-induced insertional oncogenesis.
To overcome the risk of insertional oncogenesis, a new generation of vectors using the innovative integration pattern of LVs is inherently safer than that of MLV vectors. LVs preferentially integrate throughout the transcribed portion of active genes, targeting at high-frequency genes located in the outer portion of the nucleus in proximity to the nuclear pore, with no preference for regulatory elements or specific gene categories. Early data indicate the potential of LVs as a method of gene therapy for X-SCID.
LV Development for X-SCID at Creative Biolabs
To develop safer and more effective vectors for gene therapy of X-SCID, our scientists have evaluated the self-inactivating lentiviral vectors based on the HIV virus. We have developed an LV with a short promoter, which drives a codon-optimized human IL2RG cDNA, and its efficacy and safety have been tested in vivo. Transplantation of HSPCs transduced by this vector has shown restoration of T, B, and NK cell immunity in the bone marrow and peripheral blood.
Key Features and Advantages of Our Service:
- Employing a short promoter
- Using codon-optimized human IL2RG
- Restoration of T, B, and NK cell immunity
- Safer and more effective
As a long-term expert in the field of LV development, Creative Biolabs owns lots of scientists who are proficient in LV development for X-SCID. We are confident in providing clients with the best service at the most competitive cost. Please contact us for more information and a detailed quote.
