Using the newly-developed TALEN technology, Dr. Yi has generated
novel stem cell models of Rett and Fragile X syndromes (FXS) in
human cells. Neural and glial cell types will be subjected to extensive
phenotypic characterization with respect to structure, electrophysiology,
transcription, translation and intracellular signaling. Findings
may provide critical insights into the pathogenic mechanism of Rett
and FXS in human cells, and could serve as a platform for future
therapeutic identification efforts that could lead to treatments.