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Entering the Modern Era of Gene Therapy

26 Nov 2018

Abstract excerpt

Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase. Investigators are advancing with cautious optimism that effective, durable, and safe therapies will provide benefit to patients-not only those with single-gene disorders but those with complex acquired diseases as well. While the strategies being translated...

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PMID: 30477394
DOI: 10.1146/annurev-med-012017-043332

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