Beliefs and Values About Gene Therapy and In-Utero Gene Editing in Patients with Hemophilia and Their Relatives.
Tania C Vasquez-LoarteTiffany Lin LucasJulie Harris-WaiDeborah J BowenPublished in: The patient (2021)
Patients with severe hemophilia perceive gene therapy as a potential cure, while gene editing was more controversial. Patients still have questions that remain to be answered regarding safety and efficacy that should be assessed with long-term follow up studies.