A novel treatment that uses gene therapy to correct a faulty gene in the stem cells of the recipient. Stem cells are obtained from the patient, grown and “corrected” in a laboratory, and then returned to the patient.
« Back to Glossary IndexNews
Related News

Apr 2nd, 2024
We are delighted to invite you to the upcoming FA Family Retreat, taking place at The Painted Turtle from June 21-25, 2025. The Family Retreat offers a unique opportunity for families registered with us to participate in presentations by leading...

Oct 11th, 2024
At the Fanconi Cancer Foundation (FCF), we understand the critical importance of addressing mental health alongside physical health for individuals with Fanconi anemia (FA) and their family caregivers. Recent research on the mental health challenges faced by adults with FA...

Oct 1st, 2024
Since 1991, the FA Family Retreat has been a tradition that surprises, supports, and uplifts FA families worldwide. Whether families travel from within the United States or across the globe, the retreat offers connection, educational sessions, resources, expert consultations, and...