1. Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery.

    Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery.

    Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery.

    Orphanet J Rare Dis. 2017 Apr 17;12(1):61

    Authors: Chen B, Altman RB

    Abstract BACKGROUND: Advances in next generation sequencing technologies have revolutionized our ability to discover the causes of rare genetic diseases. However, developing treatments for these diseases remains challenging.

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