Stephanie Cherqui Discusses Clinical Development of CRISPR Therapies for Rare Diseases

Professor Stephanie Cherqui, UCSD, has worked on the development of cell therapies for two rare genetic diseases, cystinosis and Friedreich’s ataxia. In this interview, Dr. Cherqui chats about her experience working on rare diseases, developing a CRISPR-edited therapy for Friedreich's ataxia, navigating regulatory challenges in cell and gene therapies, and more.