Stem Cell Gene Therapy Treatment for Cystinosis Potential Treatment for Alzheimer’s Disease

In 2003, the mission of the Cystinosis Research Foundation was to find better treatments and a cure for cystinosis. Since then, the research funded by CRF has resulted in significant advances in the field of cystinosis have been accomplished. This research has impacted other more prevalent diseases and disorders, including Danon disease and Friedreich’s Ataxia. On August 8, 2023, the University of California San Diego announced the stem cell and gene therapy treatment developed for cystinosis by Dr. Stéphanie Cherqui has the potential to treat Alzheimer’s Disease. Learn more.  Dr. Cherqui’s Alzheimer’s research was published in the prestigious journal Cell Reports on August 8, 2023. The paper is available on our Published Studies webpage.

The latest article featuring Dr. Cherqui recently appeared in the Women in RARE edition of RARE Revolution Magazine.