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Mutation-Directed Therapeutics for Neurofibromatosis Type I

29 Apr 2020

Abstract excerpt

Significant advances in biotechnology have led to the development of a number of different mutation-directed therapies. Some of these techniques have matured to a level that has allowed testing in clinical trials, but few have made it to approval by drug-regulatory bodies for the treatment of specific diseases. While there are still various hurdles to be overcome, recent success stories have proven the potential...

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PMID: 32408052
DOI: 10.1016/j.omtn.2020.04.012

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