Improving HSC engraftment by inhibiting GASP genes (SJ-16-0029)

St. Jude Reference #SJ-16-0029

Description

Hematopoietic Stem Cell (HSC) transplantation is the only curative therapy for many hematologic diseases, as well as a critical life-saving therapy following high dose chemotherapy for many non-hematopoietic cancers. Researchers at St. Jude Children’s Research Hospital have developed a way to enhance HSC transplantation by treating pre-graft cells with silencing constructs for reducing expression of GASP (G-protein coupled receptor Associated Sorting Proteins) family genes, either permanently or transiently to improve the ability of these cells to replenish the hematopoietic system of host organisms. Further, the use of GASP gene silenced umbilical cord blood-derived cells is contemplated for transplantation into HLA mismatched (allogeneic) hosts.

Benefits to this new method include:

• Accelerating reconstitution of the hematopoietic compartment to narrow the window during which patients are most susceptible to infection.
• Reducing pre-transplant conditioning to lower the risk of common long-term term side effects (secondary malignancies, adaptive immune dysfunction, growth failure, gonadal dysfunction, thyroid dysfunction).
• Improving the efficiency of umbilical cord blood (UCB)* and other HSC transplantation. (*UCB transplants tolerate greater HLA mismatch.)

Overcoming limitations on the amount of UCB cells required for HSC transplantation in adults.

Keywords

Hematopoietic Stem Cell (HSC) transplantation, GASP, G-protein coupled receptor Associated Sorting Proteins, GA2

Granted patents or published applications

US Patent application pending, published as 2019/0328791

Related scientific references

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