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Article

The Future of In Utero Gene Therapy.

Molecular diagnosis & therapy - 1 Apr 2020

Peranteau William H, Flake Alan W

Abstract excerpt

Significant advances in the safety and efficacy of gene therapy have sparked a new frontier in therapeutics for genetic diseases as evidenced by the greater than 700 active gene therapy investigational new drug applications reported by the NIH and the US Food and Drug Association. Although postnatal gene therapy trials are encouraging, limitations to effective therapy including an immune barrier and initiation of...

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Identifiers

PMID: 32020561
DOI: 10.1007/s40291-020-00445-y
PMCID: PMC9202471

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