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The Stem Cell and Gene Therapy Trial has Started!

We are thrilled to announce that following the FDA approval late last year, Dr. Cherqui’s clinical trial has officially begun at UCSD. CRF has funded Dr. Stéphanie Cherqui since 2006 issuing grants totaling over $5.4 million to support her research. The path to FDA approval was long and challenging, but Stéphanie never gave up. She remained focused on her research and committed to our community. Because of her dedication and perseverance, the treatment is now a reality and a world without cystinosis has never been more attainable.

The FDA approved Phase I and Phase II of the genetically-modified autologous stem cell transplant trial for six adults with cystinosis. The treatment involves taking hematopoietic stem cells from the patient and genetically modifying them with a lentivirus vector to insert a correct copy of the cystinosin gene. We are hopeful that this revolutionary, one-time treatment will stop the progression of cystinosis and possibly even repair some of the damage already done.

If you are an adult with cystinosis and are interested in participating in the trial or would like more information, contact Laura Hernandez lhernandez@ucsd.edu.

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