Somatic Gene Therapy Research in Pediatric Populations: Ethical Issues and Guidance for Operationalizing Early Phase Trials-Reference-Cited by-同舟云学术

Somatic Gene Therapy Research in Pediatric Populations: Ethical Issues and Guidance for Operationalizing Early Phase Trials

Published:2022-12-17 Issue:1 Volume:37 Page:17-24
ISSN:1178-2595
Container-title:Pharmaceutical Medicine
language:en
Short-container-title:Pharm Med

Author:

Bateman-House Alison^ORCID,Shah Lesha D.,Escandon Rafael,McFadyen Andrew,Hunt Cara

Funder

Parent Project Muscular Dystrophy

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Pharmacology

Link

https://link.springer.com/content/pdf/10.1007/s40290-022-00451-x.pdf

Reference35 articles.

1. American Society of Cell and Gene Therapy (ASGCT). Gene Therapy Basics. Patient Education. http://patienteducation.asgct.org/gene-therapy-101/gene-therapy-basics. Accessed 21 Nov 2022.

2. FDA approval brings first gene therapy to the United States. FDA News Release. 2017. http://fda.gov.news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states. Accessed 21 Nov 2022.

3. Maldonado R, et al. Curative gene therapies for rare diseases. J Community Genet. 2021;12(2):267–76. https://doi.org/10.1007/s12687-020-00480-6.

4. Wu AC, et al. Ending the diagnostic odyssey - is whole-genome sequencing the answer? JAMA Pediatr. 2020;174(9):821–2. https://doi.org/10.1001/jamapediatrics.2020.1522.

5. Gray SJ. Timing of gene therapy interventions: the earlier, the better. Mol Ther. 2016;24(6):1017–8. https://doi.org/10.1038/mt.2016.20.

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