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SAVE THE DATE!
Take a Swing ForeBatten 3
March 2nd, 2020
Tickets and Golf Bays on sale soon!
We are now accepting items for our silent auction. If you are interested in donating an item or becoming an event sponsor please contact Stan Kahn @ 440.725.3395
Need a hotel?
Discounted rooms are available through our room block at The Talking Stick Resort, 9800 Talking Stick Way, Scottsdale, AZ. Please call (480) 850-7777 and reference Booking ID: Forebatten 2020, ID number: 19639
What are we raising money fore?
Thanks to YOUR generosity, ForeBatten Foundation is actively sponsoring research aimed directly at CLN3-Batten disease.
GENE THERAPY
ForeBatten is sponsoring gene therapy research, focusing on pre-clinical studies of innovative AAV delivery methods to find the most effective and safe way to reach as many neuronal cells as possible. While our emphasis is CLN3-Batten Disease, this research will ultimately help many patients suffering from a wide range of neurodegenerative diseases.
ASOs
This study, with Dr. Michelle Hastings, supports the testing of an approach to correct CLN3 gene expression using a nucleic acid drug platform called antisense oligonucleotides (ASO). These molecules are designed to target CLN3 with the aim of inducing the production of functional proteins from the defective gene as a way to prevent neurodegeneration. To date, we have identified an ASO that effectively targets the defective CLN3 ex7/8 gene product in two different mouse models. Treatment of these mice with the ASO decreases cellular pathologies associated with the disease and improves motor coordination and life-span.
SMALL MOLECULES
We are also funding research in the Weimer lab, centered on identifying novel therapies for the treatment of CLN3-Batten disease including small molecules & ASO's. To better define whether selected drug targets are improving efficacy and rescuing CLN3 function, the team is using an approach called BioID, which relies of proximity-based labeling, to map the CLN3 protein interactome. In addition to mapping the protein partners of wild-type CLN3, the team will work closely with Dr. Michelle Hastings.
To learn more about the Kahn Family and Batten disease, visit www.forebatten.org
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