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Research Abstracts Online
January 2010 - March 2011

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University of Minnesota Twin Cities
Medical School
Department of Pediatrics

PI: Michael Kyba

Deriving Therapeutic Stem Cells From Embryonic Stem Cells and Skeletal Muscle Stem Cells

Embryonic stem cells are totipotent and capable of recapitulating all of the developmental events of embryogenesis. They are therefore theoretically the ideal source of cells for regenerative therapies. However, turning theory into practice is not straightforward, and very few successful models of such therapy exist. These researchers have developed one successful model, based on regulated expression of members of the Hox family of transcription factors. Current work is focused on understanding how Hox genes regulate hematopoietic stem cell self-renewal and identifying regulatory circuits under Hox control.

Certain degenerative diseases may be the result of ineffective self-renewal or differentiation of lineage specific stem cells. The researchers are particularly interested in Fascioscapulohumeral Muscular Dystrophy (FSHD), a dominant dystrophy associated with a contraction of 4q subtelomeric repeats. Although the condition is almost certainly caused by derepression of a gene in the vicinity of 4q, the protein products of candidate genes in this area cannot be detected overexpressed in patient muscle samples. Because muscle stem cells (satellite cells) are rare, proteins overexpressed specifically in satellite cells are unlikely to be identified in patient biopsies. The researchers are testing the hypothesis that a Hox gene embedded within the 4q repeats, DUX4, causes FSHD when derepressed in muscle satellite cells.

Group Members

Robert Arpke, Research Associate
Kristen England, Research Associate
Holger Fey, Research Associate
Lynn Hartweck, Research Associate
Elisabeth M. Mahen, Research Associate
Jai Richard, Research Associate
Seema Shaikh, Research Associate
Erik Toso, Research Associate
Kevin Vang Tram, Research Associate