Jun 01, 2014
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Work in my laboratory focuses on regulation of tissue-specific stem cells (hematopoietic and skeletal muscle) with a view towards ex vivo expansion and therapeutic transplantation, as well as the derivation of tissue-specific stem cells from embryonic or iPS cells. I performed seminal experiments establishing the proof of principle for hematopoietic stem cell repopulation using embryonic stem cells and maintain an active program in the development of gene-targeting / genetic correction / cell therapy models. My current major research effort focuses on FSHD and the misregulated gene responsible for this disease, DUX4. We discovered that DUX4 has pathological effects on myogenesis including evidence of competition with the myogenic master regulators Pax3 and Pax7, and proposed that a regeneration defect may be present in FSHD. With support from Friends of FSH Research, we have recently generated the first mouse model bearing human D4Z4 sequences that shows a pathological phenotype due to DUX4 expression, and have exploited this model to explore the effects of DUX4 in skeletal muscle stem cells.
Above: Regeneration by transplanted muscle stem cells. The red color indicates newly-regenerated fibers formed by transplanted muscle stem cells. Left: transplantation of control cells. Right: transplantation of cells that have turned on the DUX4 gene. Note that DUX4 reduces the regenerative potential of skeletal muscle stem cells. From Dandapat et al., 2014, Cell Reports 8:1484-1496.
Below: The Kyba Lab