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Robust preclinical evidence in somatic cell genome editing: a key driver of responsible and efficient therapeutic innovations.

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Somatic cell genome editing (SCGE) shows significant promising for therapeutic innovation. This study demonstrates that the majority of 46 preclinical SCGE studies discussed in reviews as particularly promising for clinical… Click to show full abstract

Somatic cell genome editing (SCGE) shows significant promising for therapeutic innovation. This study demonstrates that the majority of 46 preclinical SCGE studies discussed in reviews as particularly promising for clinical translation do not report on key elements for robust and confirmatory research practices: randomization, blinding, sample size calculation, data handling, pre-registration, multi-centric study design, and independent confirmation. We present the here-examined reporting standards and the new National Institutes of Health (NIH) funding criteria for SCGE research as a viable solution to protect this promising field from backlashes. We argue that the implementation of the novel methodological standards provides the opportunity for SCGE research to become a lighthouse example for trustworthy and useful translational research.

Keywords: genome editing; cell genome; research; somatic cell

Journal Title: Drug discovery today
Year Published: 2021

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