It’s a “Great Idea” Dr. Sacco!

Written by 
Susan Gammon
Alessandra Sacco

Alessandra Sacco, Ph.D., associate professor in the Development, Aging, and Regeneration Program has been awarded a CIRM “Great Idea.” The award was one of six that are part of CIRM’s Discovery Inception Program to provide funding for great ideas that could impact stem cell research but need funding support to test those ideas. 

Sacco’s proposal, “Generation of expandable, self-renewing muscle stem cells for Duchenne Muscular Dystrophy” will test a new method to boost the supply of cells that help build skeletal muscle—a step toward future therapies for this devastating disorder that affects 1:3500 males.

Although the genetic defect responsible for the disease, the lack of a functional dystrophin protein, has been known for years, still no cure is available to patients.

Patients develop paralysis, cardiorespiratory failure, and death in their early 20s.

The progression of the disease is associated with a loss of muscle regenerative potential, as muscle stem cells (MuSC) become functionally exhausted. MuSCs give rise to skeletal muscle cells, and are involved in the normal growth of muscle, as well as regeneration following injury or disease.

“Our goal is to develop a method whereby self-renewing human MuSCs could be expanded in vitro and then used for “disease in a dish” modeling and drug screening as well as transplanted to provide a supply of cells that self-renew long-term.

“CIRM’s support will help our research team gather the data necessary to apply for larger funding opportunities, from CIRM and other institutions, in the future,” says Sacco.

What is CIRM?

CIRM, California’s stem cell institute, was created by the voters of California in 2004 when they passed Proposition 71. The institute funds stem cell research at institutions and companies throughout California with the goal of accelerating treatments to patients with unmet medical needs.