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Ucsf faculty receive $13.7m from cirm for stem cell research

Five UCSF stem cell scientists have received New Faculty grants from California Institute for Regenerative Medicine, support that'll allow them to pursue lines of investigation ultimately aimed at developing treatment strategies for such conditions as cancer, heart disease, tooth regeneration, liver disease and amyotrophic lateral sclerosis.

The UCSF grants, which range from about $2 million to $3 million each, total $13. 7 million and are intended to support scientists in critical early stages of their careers as independent investigators establishing their own laboratories and research programs. CIRM will provide salary and research support for up to five years, creating a stable environment for faculty to build innovative and robust stem cell research programs.

" These grants are a tremendous boon to investigators, but also to stem cell field overall, " says Arnold Kriegstein, MD, PhD, director of the UCSF Institute for Regeneration Medicine. " They will fuel research of some of the most creative stem cell scientists in the world, allowing them to pursue significant scientific challenges they have set for themselves. "

The grants, announced today, are latest round of research funds awarded by the Independent Citizens Oversight Committee, which oversees CIRM. The ICOC awarded $59 million in New Faculty grants to research universities throughout California.

The UCSF researchers will use funds to explore fundamental questions about the way in which embryonic stem cells function, how they are regulated and what molecular steps lead them to specialize as specific types of mature cells, such as liver cells. In each case, they plan to use knowledge they gain to explore cell-replacement therapies or identify potential drugs to treat diseases.

The UCSF faculty recipients, their research areas, and grant amounts are:

• Robert Blelloch, MD, PhD - He'll study molecules known as small RNAs, which he hypothesizes play a key role in controlling expression of embryonic stem cells. Understanding mechanisms that determine and maintain potential of stem cells will allow medical and pharmaceutical community to improve efficiency with which they can produce embryonic stem cell lines, as well as quality control of resulting cells, he says. ( $3, 029, 897 grant )

• Ophir Klein, MD, PhD - He will explore molecular processes that regulate behavior of dental stem cells in mice, with the goal of promoting tooth regeneration and craniofacial abnormalities in humans. As signaling pathways are similar to those of liver and other organs, his work will shed light on regeneration of these organs, as well. His ultimate goal, he says, is to help lay groundwork for growing human teeth and help advance efforts to regenerate larger organs. ( $3, 075, 251 grant )

• Emmanuelle PasseguÃ, PhD - She will study mouse blood stem cells to investigate how they thrive and, at times, accumulate damage that leads to cancer. Her goal is to identify molecular targets for preventing therapy-related organ damage or secondary cancers. She also will use her mouse model of human leukemia to study what molecular changes lead to cancer stem cell formation and function during disease development. The goal of this work would be to design novel therapies that target cancer stem cells while sparing normal stem cells. ( $2, 274, 368 grant )

• Jeremy Reiter, MD, PhD - He will study genes that contribute to development of ALS and other forms of neurodegenerative motor diseases. Using a mouse model he developed, his team will examine the impact of mutant genes associated with diseases on mouse embryonic stem cells, in order to identify the way in which they actually kill cells. Diseased motor neurons grown in cell culture dishes could be quickly and efficiently screened with potential drugs to discover agents that slow, halt or reverse cellular damage, he notes. ( $2, 259, 092 grant )

• Holger Willenbring, MD - He'll investigate the genes that establish function of early-stage liver cells. His goal is to use this knowledge to prompt human embryonic stem cells to specialize as early-stage liver cells in culture dish. If he succeeds, he'll transplant cells into a mouse model of human liver disease. This would be proof of principle for potential of embryonic stem cells for liver cell therapy, he says. ( $3, 032, 510 grant )

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UCSF is a leading university that advances health worldwide by conducting advanced biomedical research, educating graduate students in the life sciences and health professions, and providing complex patient care.

ADDITIONAL DETAILS FOLLOW:

Click here for descriptions of each grant award.

• Robert Blelloch, MD, PhD, is UCSF assistant professor of urology. " Mechanisms of small RNA regulation in early embryonic development. " ( RN2-00906-1 )

• Ophir Klein, MD, PhD, is UCSF assistant professor of orofacial sciences, pediatrics, and human genetics. " Laying groundwork for building a tooth: analysis of dental epithelial stem cells. " ( RN2-00933-1 )

• Emmanuelle PasseguÃ, PhD, is UCSF assistant professor of medicine. " Mechanisms Underlying Responses of Normal and Cancer Stem Cells to Environmental and Therapeutic Insults. " ( RN2-00934-1 )

• Jeremy Reiter, MD, PhD, is UCSF assistant professor of biochemistry and biophysics. " High throughput modeling of human neurodegenerative diseases in embryonic stem cells. " ( RN2-00919-1 )

• Holger Willenbring, MD, is UCSF assistant professor of surgery. " Molecular dissection of adult liver regeneration to guide the generation of hepatocytes from pluripotent stem cells. " ( RN2-00950-1 )

RELATED LINKS: ~ UCSF Institute for Regeneration Medicine: http: //irm. ucsf. edu/ ~ California Institute for Regenerative Medicine: http: //www. cirm. ca. gov/ ~ University of California, San Francisco: http: //www. ucsf. edu/

Source: Jennifer O'Brien University of California - San Francisco

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