Professor Kirill Martemyanov at University of Florida Scripps Institute for Biomedical Innovation and Technology will help advance scientific understanding about the mechanisms of dystonia. Many GNAO1 patients suffer from dystonia, commonly known as involuntary muscle movements. Bow Foundation funding will allow Martemyanov to use a mouse model to shine light on the impact of GNAO1 on dystonia and brain signals while also testing possible treatment strategies.
Funded by The Bow Foundation
Dr. Jennifer Friedman of the University of California San Diego and Rady Children’s Hospital is partnering with the n-Lorem Foundation to support the administration of an experimental antisense oligonucleotide (ASO) medicine that targets the GNAO1 gene. Funding from the Bow Foundation will help Dr. Friedman collect and evaluate the clinical observations of this cutting-edge treatment, including changes in baseline over time and data from predetermined outcome measures. This preclinical work will allow the research team to determine if ASO treatments for other GNAO1 patients are a viable approach for other patients.
Funded by The Bow Foundation
Dr. Harald Mikkers of Leiden University Medical Center in the Netherlands will work to advance and improve a state-of-the-art personalized medicine tool for GNAO1. He will use funding from the Bow Foundation to create a validated stem cell GNAO1 model that opens the doors to various drug screening efforts. The work will investigate how GNAO1 impacts neurons and evaluate the suitability of the iPSC-based model for testing of therapeutics and drug responses.
Funded by The Bow Foundation
After this act of love and kindness, we knew that we needed to fundraise and pay it forward.
Our first fundraiser was a resounding success, and this is the write up about it in our local paper from prior to the event.
The follow up on our story in the local paper. We could not be more grateful.
This news article comes out of Canada about some very promising research directly related to GNAO1.
This links to a medical journal article released in 2017 that takes a deep dive into what GNAO1.
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