The Lived Experience of Pediatric Gene Therapy: A Scoping Review

• Published in: Human gene therapy Vol. 34; no. 23-24; p. 1180
• Main Authors: Kimberly, Laura, Hunt, Cara, Beaverson, Katherine, James, Emma, Bateman-House, Alison, McGowan, Richard, DeSante-Bertkau, Jennifer
• Format: Journal Article
• Language: English
• Published: United States 01.12.2023
• Subjects: Adult; Caregivers - psychology; Child; Clinical Trials as Topic; Genetic Therapy; Humans; Parents - psychology; clinical trial ethics; pediatric gene therapy; patient experience; pediatric clinical trials; lived experience

Little is known about patients' and families' lived experiences of participating in pediatric gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of indications--including rare and ultra-rare diseases--which vary in severity and in the availability of...