Patent ID: 8257941

Claim:
A method for identifying an agent that corrects a phenotype associated with a health condition or a predisposition for the health condition, comprising: (i) contacting a first population of isolated cells differentiated from a human induced pluripotent stem cell line, wherein said cells exhibit a phenotype associated with the health condition or predisposition for the health condition, with a candidate agent; (ii) contacting a second population of isolated cells differentiated from the human induced pluripotent stem cell line, wherein said cells exhibit a phenotype associated with the health condition or predisposition for the health condition, with a negative control agent; (iii) assaying a phenotype in the first population and second population after the contacting steps; and (iv) identifying the candidate agent as correcting the phenotype if the assayed phenotype of the first population after the contacting step is closer to a normal phenotype than the phenotype of the second population after the contacting step; wherein one or more cells differentiated from the induced pluripotent stem cell line comprise an exogenous Oct3/4 gene, an exogenous Sox2 gene, and an exogenous Klf4 gene; wherein the cells in the first and second populations of human induced pluripotent stem cells: (a) comprise at least one endogenous allele associated with the health condition or the predisposition for the health condition; or (b) are generated from a subject suffering from the health condition or the predisposition for the health condition.