La Jolla, CA -- The Salk Institute for Biological Studies has been awarded a $6.6 million grant – the largest single award in the latest competition -- by the California Institute Regenerative Medicine (CIRM) for research aimed at translating basic science into clinical cures. The funds are part of $67.7 million Early Translational Grants CIRM provided to 15 research organizations on Wednesday.

Led by Salk scientists Drs. Inder Verma and Juan Carlos Izpisúa Belmonte, the research will focus on developing treatments for Fanconi anemia and X-Linked Severe Combined Immunodeficiency (X-SCID), more commonly known as the "bubble boy" disease. Both are diseases of the blood and each is caused by the mutation in a single gene.

Using pioneering gene therapy and stem cell reprogramming techniques developed by Drs. Verma and Belmonte, they will work with mouse models that mimic each of the diseases. The human hematopoietic (blood precursor) stem cells used in their work are derived from patient hair follicle cells that have been induced into a pluripontent stem cell state (iPS) and corrected of its defective gene. The genetically corrected cells will then be coaxed back into the cells that form the blood and immune systems and used for transplant therapy.

"Working with human patient and disease-specific cells will help us demonstrate the feasibility and evaluate the safety in a pre-clinical setting to advance these techniques, which combine the latest developments in regenerative medicine and gene therapy," said Verma, a professor in the Laboratory of Genetics. "This work will also benefit the successful stem cell-based therapies for many other diseases like Parkinson's and diabetes."

"The opportunity to work with human cells from patients with these diseases will further demonstrate why we believe these cells are perfect candidates for transplantation therapy," said Belmonte, a professor in the Gene Expression Laboratory. "The chances of rejection are drastically reduced when the cells are derived from the patients themselves."

The 15 Early Translational grants approved by the CIRM's board were awarded to 13 not-for-profit and two for-profit organizations. They are intended to either lead to a drug candidate for an unmet medical need or address a bottleneck in the development of new therapies.

"With these Early Translational grants CIRM has taken the first step in funding translational research that will be critical for the development of future therapies," said Alan Trounson, CIRM president. "These grants are an important part of CIRM's strategy to fund the best basic research and then bring the results of that work to patients."

About the Salk Institute for Biological Studies:
The Salk Institute for Biological Studies is one of the world's preeminent basic research institutions, where internationally renowned faculty probe fundamental life science questions in a unique, collaborative, and creative environment. Focused on both discovery and mentoring future generations of researchers, Salk scientists make groundbreaking contributions to our understanding of cancer, aging, Alzheimer's, diabetes, and cardiovascular disorders by studying neuroscience, genetics, cell and plant biology, and related disciplines.

Faculty achievements have been recognized with numerous honors, including Nobel Prizes and memberships in the National Academy of Sciences. Founded in 1960 by polio vaccine pioneer Jonas Salk, M.D., the Institute is an independent nonprofit organization and architectural landmark.