Research
Gene therapy involves gene transfer into stem cells but is hindered by low gene expression levels. Stem cells silence retrovirus vectors by compacting DNA into inaccessible chromatin structures. To overcome this obstacle will require the development of viral vectors that escape gene silencing and the identification of DNA regulatory elements that direct high levels of transgene expression. We develop vectors to specifically mark stem cells or their differentiated progeny. In particular, we use these vectors to reprogram somatic cells to induced pluripotent stem (iPS) cells. We also model disease by generating and studying iPS cells derived from patients.
Major research activities in the Ellis Lab
iPS cells are being generated from patients to study neuropsychological disorders including autism and schizophrenia. In addition we use iPS cells to model cystic fibrosis and cardiac disorders.
Retrovirus silencing pathways that compact chromatin in mouse embryonic stem cells and iPS cells are being identified. Retrovirus vectors that resist silencing are being enhanced with insulator elements that block the spread of silent chromatin.
Retrovirus and lentivirus vectors that transfer specific MeCP2 isoforms into Neural Stem Cells are being developed for gene therapy of Rett Syndrome.