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What is the context of this research?
This project will determine whether anle138b can delay the onset of symptoms and extend survival in a mouse model of A117V Gerstmann-Straussler-Scheinker syndrome, a genetic prion disease.
update: We have now announced a study design and budget for studies of this compound in mouse models of fatal familial insomnia (FFI) caused by the D178N 129M mutation, and Creutzfeldt-Jakob disease (CJD) caused by the E200K mutation. Together with the original GSS project, these studies will determine whether anle138b is effective against a range of different genetic mutations that cause prion disease.
update #2: We have now met all three goals! In our final day we will raise funds toward our many other research goals. We want to understand how prion protein is regulated in the cell, use gene silencing approaches to reduce it, develop new assays, investigate other potential drug candidates, and much more!
What is the significance of this project?
anle138b is the most promising anti-prion compound yet discovered, but to date it has only been tested in vivo against one prion strain. This project will determine its efficacy against an entirely different strain of prions, in a model that closely mimics the pathology of a human prion disease.
Prion diseases are extremely rapid, with mortality following only months after initial symptoms. However, many carriers of genetic prion diseases - including us - have undergone predictive genetic testing and know their status decades before the onset of any symptoms. Genetic prion diseases provide an opportunity for early intervention and a potentially large therapeutic effect.
What are the goals of the project?
Funds for this project will be spent on mouse space, labor and equipment for a therapeutic trial in GSS mice at Dr. James Mastrianni's facility at University of Chicago. The experimental compound anle138b will be provided for free by the drug's inventors, Dr. Armin Giese and Dr. Christian Griesinger.
update: Funds raised beyond our original goal will go towards trials of anle138b against fatal familial insomnia and E200K Creutzfeldt-Jakob disease led by Dr. George Carlson at McLaughlin Research Institute. A detailed budget for these additional studies can be found in this blog post. Each study will cost $4,400 and they are represented by Goal 2 and Goal 3 above.