Rational Design of Effective Therapeutics for Treatment of the 1282X CFTR Disorder

Awardee: Cooperman

Institution: University of Pennsylvania

Award Amount: $53,000

Funding Period: January 1, 2017 - December 31, 2017

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Gene-edited stem cells for modeling rare mutations in cystic fibrosis (CF)

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The efficacy of guaifenesin in the treatment of adult polyglucosan body disease