Goodlett, Charles R PHD

INCLUDE Grants​

Strategies for treatment of Down syndrome: Identifying age- and sex-specific developmental windows using inducible genetic reduction of Dyrk1a

Goodlett, Charles RRoper, Randall J

The proposed research is relevant to public health because it uses multi-disciplinary innovative approaches to create new scientific paradigms of trisomic gene expression, and establish improved approaches to translate these therapies from preclinical to clinical trials. Inducible genetic approaches in DS mouse models will target the amplified expression of Dyrk1a during critical periods of brain and bone development in trisomic mice, testing the hypothesis that normalization of trisomic gene expression before—but not after—tissue-specific periods of gene dysregulation will correct cognitive and skeletal deficits. The proposed research is relevant to the part of NICHD’s mission to perform research to insure that that all children have the chance to achieve their full potential for healthy and productive lives, free from disease or disability.