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Article

Are we prepared to deliver gene‐targeted therapies for rare diseases?

24 Jan 2023

Abstract excerpt

The cost and time needed to conduct whole-genome sequencing (WGS) have decreased significantly in the last 20 years. At the same time, the number of conditions with a known molecular basis has steadily increased, as has the number of investigational new drug applications for novel gene-based therapeutics. The prospect of precision gene-targeted therapy for all seems in reach… or is it? Here we consider practical...

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PMID: 36691939
DOI: 10.1002/ajmg.c.32029

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