The following are lay summaries from research projects that NAF was able to fund because of generous contributions from our donors. All of these research summaries are of grants funded by NAF for fiscal year 2018. Thank you to each of you who made a donation to last year’s Research Drive “Proud Past… Focused Future.”
Unless you are a scientist, these research summaries can seem like “Greek” to you, however, it does demonstrate the complexity of science, particularly neuroscience. These summaries were submitted directly from the researchers. While they may be difficult to read, we at NAF think it is important to keep you up-to-date on the science that your membership and donations support.
There are currently no therapies that delay onset or progression of spinocerebellar ataxia. In earlier work, we showed that gene silencing approaches or gene over-expression approaches delivered individually had a profound positive impact on disease readouts in two animal models of spinocerebellar ataxia type 1 (SCA1). Additionally, our gene silencing therapies in SCA1 mice reversed behavioral deficits and neuropathology, even when delivered after onset. These data are the foundation for future clinical application of gene silencing studies in SCA1 patients. Here, we propose to expand and improve on this work by testing a combinatorial approach. The goal of this newer approach is to reduce the overall dose of material needed to achieve therapeutic benefit. If our tests in mice are successful, we will seek additional funding to move this forward to SCA1 patients.
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