Pattern of Small Genetic Factors Found to Characterize Embryonic Stem Cells

Researchers at The Scripps Research Institute discovered that human embryonic stem cells have a very specific signature when it comes to the regulators of their genes. MicroRNAs are very small, naturally occurring bits of genetic material. They don't code for specific proteins like genes do, but they regulate the activity of genes and turn on and off their protein production. In embryonic stem cells microRNAs are actively preventing the production of proteins that tell cells to differentiate into specific heart or bone tissue, for example, but are pushing hard on genes that result in self-renewal. The team hopes to use these microRNAs to reprogram any type of cell to become as pluripotent as embryonic stem cells and to do it more safely than current reprogramming called iPS.

Stem Cells: June, 2008
CIRM funding: Louise Laurent (T1-00003)

Related Information: Press release , The Scripps Research Institute

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

Method Produces Nerve Cells More Quickly

Researchers at the Burnham Institute for Medical Research have developed a new way of quickly maturing embryonic stem cells into neural cells. Other research groups have worked out lab conditions that encourage embryonic stem cells to mature into various types of nerve cells, but those methods were slow and resulted in early stage nerve cells that were more likely to cause tumors when transplanted into mice. This new method could speed work by researchers who are trying to develop therapies for diseases of the nervous system. As an additional benefit, this work showed that some previously overlooked genes are worth studying as potential regulators of embryonic stem cell maturation.

Cell Death and Differentiation: March 13, 2009
CIRM authors: R Bajpai (T2-00004), Stuart Lipton (RC1-00125), Alexi Terskikh (RS1-00466)

Related Information: Press release, Burnham Institute for Medical Research, Lipton bio, Terskikh bio

Genetic Factor Influences Heart Muscle Formation from Embryonic Stem Cells

Researchers at the Gladstone Institute for Cardiovascular Disease discovered how two specific tiny genetic factors called microRNAs influence the differentiation of embryonic stem cells into heart muscle. They found that the factors not only drive the versatile cells to become heart, but also actively prevent them from becoming other tissue such as bone adding to their potential to make therapy more specific and targeted for patients.

Cell Stem Cell: March 6, 2008
CIRM funding: Kathey Ivey (T2-00003), E. Hsiao (T2-00003), Deepak Srivastava (RC1-00142)

Related Information: Press release, Gladstone Institute of  Cardiovascular Disease,Srivastava bio

Validation of Technique Inducing Skin Cells to become Pluripotent Stem Cells

Researchers at UC, Los Angeles succeeded in inducing skin cells to become pluripotent cells with genetic featured very much like embryonic stem cells. They verified work published during the completion of their project, which showed that the introduction of four specific genetic factors is sufficient to induce differentiated adult cells into reverting to an embryonic stem cell-like state. This was critical validation of a procedure that could lead to a new way of developing personalized cell lines for therapy.

Proceedings of the National Academy of Sciences: February 26, 2008
CIRM funding: Rupa Sridharan (T1-00005)

Related Information: Press release, The Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Lowry lab page

Mutation Causing Cardiomyopathy Validated in Mouse Embryonic Stem Cells

Researchers at UC, Irvine used mouse embryonic stem cells to demonstrate that a specific mutation can cause cardiomyopathy, with a thickened heart wall, in the mouse. The team looked at the small DNA molecule located outside of the nucleus, so-called mitochondrial DNA, which we all inherit exclusively from our mothers. They also discovered that severe mutations in this mitochondrial DNA are readily eliminated from the mouse germ line in just four generations. They expect the method they used to become a robust research tool to study the impact of mutations on stem cells.

Science: February 15
CIRM-funded authors: Weiwei Fan (T1-00008), Douglas Wallace (RC1-00353)

Related Information: Press release, Sue and Bill Gross Stem Cell Research Center,

Genes Identified as Unique to Specialized Colon Cells

Researchers at University of California, San Francisco found nearly a thousand genes that are expressed differently in different parts of the colon. The colon is constantly renewed via its own stem cells and understanding how these genes are expressed differently as the cells specialize will help understand what happens when this goes wrong as in colon cancer.

Proceedings of the National Academy of Sciences: September 25, 2007
CIRM-funded author: Cynthia Kosinski (T1-00002)

Related Information: UCSF Institute for Regeneration Medicine