Article
Applying the logic of genetic interaction to discover small molecules that functionally interact with human disease alleles.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2015
Brettman Ari D, Tan Pauline H, Tran Khoa, Shaw Stanley Y
Abstract excerpt
Despite rapid advances in the genetics of complex human diseases, understanding the significance of human disease alleles remains a critical roadblock to clinical translation. Here, we present a chemical biology approach that uses perturbation with small molecules of known mechanism to reveal mechanistic and therapeutic consequences of human disease alleles. To maximize human applicability, we perform chemical...
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