Prion disease can be effectively treated or delayed in animals by reducing the amount of prion protein in the brain. Recent data indicate that potent, long-lasting reduction of a specific protein in the brain can be achieved by zinc finger proteins (ZFPs) engineered to turn off a gene by binding the DNA and preventing its transcription into RNA. This offers a potential new therapeutic strategy in prion disease. We will test the ability of ZFPs targeting the prion protein gene to lower brain prion protein levels and prevent or treat disease in prion-infected mice.
About the Researcher:
Sonia Vallabh co-runs a prion research laboratory at the Broad Institute of MIT and Harvard along with her husband, Eric Minikel. She earned her PhD in Biological and Biomedical Sciences from Harvard Medical School in 2019. Sonia and Eric left their previous careers to devote their lives to biomedical research after learning in 2011 that Sonia had inherited from her mother a mutation that causes genetic prion disease. At the Broad Institute, Sonia works on prion disease drug discovery, with a focus on lowering the amount of prion protein in the brain. She also works on biomarkers and other tools to enable drugs to be tested for their ability to prevent or delay, as well as treat, prion disease.
Dr. Vallabh is the recipient of:
- The CJD Foundation Grant, contributed by the Families of the CJD Foundation
- The Diana Hunter Memorial Grant, contributed by The Hunter Family and Byron Jones
- The Cheryl Molloy Memorial Grant, contributed by Tim Molloy and Family
- The Lynda Morris Memorial Grant, contributed by David Morris and Family
- The Daniel Nugent Memorial Grant, contributed by the Highman and Nugent Families
- The Eugene A. Riedel Memorial Grant, contributed by Jacqueline (Toby) Riedel
- The Jeffrey A. Smith Memorial Grant, contributed by The Jeffrey and Mary Smith Family Foundation
- The Strides for CJD Grant, contributed by the Families of the CJD Foundation
- The Walter Williams Memorial Grant, contributed by Susan and Kyle Williams and the Aarons Law Firm