The Hyper IgM Foundation Blog 

For Immediate Release

July 5, 2017

Hannah Ross

The Hyper IgM Foundation Presents $5,000 Grant to Dr. Caroline Kuo at UCLA

NEW YORK, New York—The Hyper IgM Foundation is delighted to announce it has awarded $5,000 to Dr. Caroline Kuo and the Regents of the University of California, Los Angeles, to support research in connection with Site-Specific Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome.

These funds will support the continued work in search of curative treatment for X-Linked Hyper IgM Syndrome (X-HIGM). Currently, the only known cure for X-HIGM is a stem cell transplant. There are no curative treatment options, however, for those X-HIGM patients without an HLA match or those with chronic infections or other conditions that make allogeneic stem cell transplant too risky. Graft rejection and graft-versus-host disease in patients following a stem cell transplant remain major issues for patients. Dr. Kuo plans to use the grant to further perfect the use of stem cell manipulation without the need for a donor. What this means is a transplant of the patient’s own, gene-corrected stem cells after having undergone site-specific stem cell gene therapy. In turn, there should be no rejection from the host, being that the cells are recognized as same DNA.

In the next year (July 1, 2017 to June 30, 2018), the funding from the grant will help cover continued work and ensure that site-specific gene editing in human stem cells can be long-lasting and self-renewing. In other words, if doctors can fix the defective gene in the stem cell and give it back to the patient, the hope is that the now-corrected stem cell will give rise to normal T-cells that efficiently express CD40 Ligand for the rest of the patient’s life — leading to full immune response for the individual.

The proposal reads, “These experiments set the foundation for a promising therapy for patients with X-linked Hyper IgM Syndrome.” Hyper IgM affects only 1 in 1,000,000 individuals. The life-threatening genetic mutation severely compromises the immune system and its ability to produce responses to pathogens such as bacteria and viruses. The Hyper IgM Foundation’s mission is to improve the treatment, quality of life and long-term outlook for children and adults living with Hyper IgM.

The Foundation gives families coping with Hyper IgM the opportunity to connect with other patient families to gain both knowledge and support.

The Hyper IgM Foundation also fundraises all year long in order to fund progress in research for a cure. Last year alone, the foundation raised over $40,000. The goal is to see this gene-editing treatment moved forward into clinical trials, and that is where help is needed.

For more information about Hyper IgM Syndrome or to support the cause please visit www.hyperigm.org/donate.

If you have any questions or interest in supporting our foundation, please email [email protected].