"Addressing the Crisis: Experts Seek Solutions for Gene Therapy's Challenges"

The field of gene therapy is facing a crisis as researchers struggle to get their treatments approved for rare diseases due to the small number of patients. Jennifer Puck, an immunologist at the University of California, San Francisco, has successfully treated 10 children with a gene therapy for Artemis-SCID, but is unsure about how to proceed with getting her drug approved. The low number of patients makes it difficult for companies to generate profits or conduct the necessary studies required by regulators.