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UC San Diego Launches Gene Therapy Initiative With $5M Philanthropic Gift

NEW YORK – The University of California, San Diego on Tuesday launched an initiative within which researchers will develop treatments for rare genetic diseases.

The UC San Diego Gene Therapy Initiative has received $5 million in philanthropic funding from the Nancy and Geoffrey Stack Foundation. The Stacks' daughter, Natalie, was diagnosed as a newborn with cystinosis, a rare genetic condition that results in damage to the kidneys, liver, and other organs. With previous funding from the Stacks, researchers at UC San Diego including Stephanie Cherqui, a professor of pediatrics at the university and chair of the Cystinosis Stem Cell and Gene Therapy Consortium, developed a slow-release version of a medication for cystinosis patients and, more recently, a stem cell gene therapy approach that has benefited numerous patients, including their daughter, who is now 32 years old.

With the latest infusion of funds from the Stacks, UC San Diego hopes to bring together the expertise, resources, and knowledgebase to discover and develop new therapies, including ex vivo stem cell therapies and in vivo treatments, which can benefit patients with rare conditions caused by mutated or missing genes. The Gene Therapy Initiative will be led by Cherqui and Alysson Muotri, a professor in the departments of pediatrics and cellular and molecular medicine at UC San Diego's School of Medicine.

"We are in a time like no other to advance the development of new gene-based therapies to benefit children and adults with genetic disorders," Cherqui said in a statement. "We are already finding success in treating some diseases with these methods, and this gift will help us expand that scope."