Waddington, Simon N;
Peranteau, William H;
Rahim, Ahad A;
Boyle, Ashley K;
Kurian, Manju A;
Gissen, Paul;
Chan, Jerry KY;
(2023)
Fetal gene therapy.
Journal of Inherited Metabolic Disease
10.1002/jimd.12659.
(In press).
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Abstract
Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene therapy was, to quote the Gartner hype cycle, at its “peak of inflated expectations.” Gene therapy was still an immature field but over the ensuing decade, it matured and is now a clinical and market reality. The trajectory of treatment for several genetic diseases is toward earlier intervention. The ability, capacity, and the will to diagnose genetic disease early—in utero—improves day by day. A confluence of clinical trials now signposts a trajectory toward fetal gene therapy. In this review, we recount the history of fetal gene therapy in the context of the broader field, discuss advances in fetal surgery and diagnosis, and explore the full ambit of preclinical gene therapy for inherited metabolic disease.
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