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Genetic Differences Could Impact Efficacy and Safety of CRISPR

06:04 EST 12 Dec 2017 | Genetic Engineering News

Studies by researchers in the U.S. and Canada suggest that small differences in the genetic codes of individuals could have a significant impact on the efficacy, side effects and safety of treatments based on CRISPR/Cas9 gene editing. The teams at Harvard Medical School, Boston Children’s Hospital, and the Université de Montréal say their findings suggest that a one size fits all approach to therapeutic gene editing may not be equally effective for all patients, and that the editing technologies may need to be adjusted to each patient’s genome sequence to maximize efficacy and also prevent potentially harmful off-target effects. "Humans vary in their DNA sequences, and what is taken as the 'normal' DNA sequence for reference cannot account for all these differences," says Stuart Orkin, M.D., of Dana-Farber Boston Children's Cancer and Blood Disorders Center. "We recommend that common variation be taken into account in designing targeting systems ...

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