Sustained siRNA production from human MSC to treat Huntingtons Disease and other neurodegenerative disorders

The Early Translational Awards, which are funded annually, either lead to a drug candidate for an unmet medical need or address a bottleneck in the development of new therapies. The 16 Early Translational I awards worth $71,570,007 were awarded on 4/29/09. You can learn more about the awards by reading the RFA or reading the press release.

Public Abstract (provided by applicant)

One in every ten thousand people in the USA have Huntington's Disease, and it impacts many more. Multiple generations within a family can inherit the disease, resulting in escalating health care costs and draining family resources. This highly devastating and fatal disease touches all races and socioeconomic levels, and there are currently no cures. Screening for the mutant HD gene is available, but the at-risk children of an affected parent often do not wish to be tested since there are currently no early prevention strategies or effective treatments. HD is a challenging disease to treat. Not only do the affected, dying neurons need to be salvaged or replaced, but also the levels of the toxic mutant protein must be diminished to prevent further neural damage and to halt progression of the movement disorders and physical and mental decline that is associated with HD. Our application is focused on developing a safe and effective therapeutic strategy to reduce levels of the harmful mutant protein in damaged or at-risk neurons. We are using an RNA interference strategy

Statement of benefit to California (provided by applicant)

It is estimated that one in 10,000 CA residents have Huntington