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Article

Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing.

Molecular genetics and metabolism - 1 Jan 2000

Domm Jakob M, Wootton Sarah K, Medin Jeffrey A, West Michael L

Abstract excerpt

Gene therapy is the delivery of a therapeutic gene for endogenous cellular expression with the goal of rescuing a disease phenotype. It has been used to treat an increasing number of human diseases with many strategies proving safe and efficacious in clinical trials. Gene delivery may be viral or non-viral, performed in vivo or ex vivo, and relies on gene integration or transient expression; all of these...

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PMID: 34340879
DOI: 10.1016/j.ymgme.2021.07.006

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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