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Michigan Researchers Create Stem Cell Lines for Hemophilia B

Investigators from the University of Michigan (U-M) in Ann Arbor have created six new embryonic stem cell lines for US-based research. Two of the new lines carry the genetic mutation linked to hemophilia B, the first of its kind.


Stem cells are unspecialized cells that can renew themselves for prolonged periods. They can also develop into many different cell types, making them a potentially renewable source of replacement cells that could be used in the future to treat many conditions. The creation of such stem cell lines will open the door to an enhanced understanding of the origin and progression of congenital disorders, and hopefully to new therapies based on those findings.


U-M, which has been creating stem cells for more than three years and is a leader in the field, now has eight lines available for federally funded researchers nationwide. Access to embryonic stem cells was first made possible in Michigan in November 2008 when voters approved a state constitutional amendment allowing researchers to generate new lines from unused embryos donated by fertility clinics. Instead of discarding the embryos, which in some instances carry the genes responsible for inherited disorders, scientists now have a new approach for advancing research.


The amendment also led to a partnership between U-M’s Consortium for Stem Cell Therapies (CSCT) and Detroit-based Genesis Genetics, leading global provider of pre-implantation genetic diagnosis (PGD) testing, which identifies days-old embryos carrying disease-causing genetic mutations. The partnership offers patients the option of donating embryos that test positive for a genetic disorder to the CSCT.


In addition to hemophilia B, U-M now has lines available to study a number of conditions such as Huntington’s disease, a fatal brain disorder, and hypertrophic cardiomyopathy, a heart condition associated with sudden death in athletes and others.


“Our last three years of work have really begun to pay off, paving the way for scientists worldwide to make novel discoveries that will benefit human health in the near future,” said Gary Smith, PhD, who derived the lines. He is co-director of CSCT.


Source: The Detroit News, June 14, 2012

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