First clinical Trial Begins for a Therapy Enabled By CIRM Funding

Researchers at UC, San Diego verified a suspect gene mutation in blood-forming stem cells was by itself necessary and sufficient to cause a class of severe blood diseases called myeloproliferative disorders. They then worked with a team of researchers from other academic institutions and from the San Diego pharmaceutical company TargeGen to conduct animal tests of a compound TargeGen had already isolated and shown to inhibit that same genetic pathway. As a result of this broad collaboration, human clinical trials for this potential therapy began in February, 2008.



CIRM funding: Catriona Jamieson

Related Information: UC San Diego press release, UCSD Stem Cell Initiative

© 2013 California Institute for Regenerative Medicine