Your generosity at work
As a foundation, we are incredibly blessed to have a wonderful community of support embracing our mission and cause. With each and every thoughtful donation, your dollars are put to work funding a variety of important projects. Ranging from therapies for a cure to improving quality of daily life, our goal is to diversify and attack the problem from multiple angles, while being guided by the brightest scientific minds in the rare disease space.
Below you will find a list of currently funded projects and a brief description of each. Thank you for your support.
This study provides support to our testing of an approach to correct CLN3 gene expression in CLN3 Batten disease 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 CLN3Δex7/8 gene product in two different mouse models of CLN3 Batten disease. Treatment of these mice with the ASO decreases cellular pathologies associated with the disease and improves motor coordination and life-span. A manuscript reporting these results and acknowledging the Fore Batten Foundation for support of studies in the later stages of this study has been submitted for publication. On-going studies are investigating the long-term efficacy of the ASOs as well as testing ASO approaches for treating patients with CLN3 Batten disease caused by CLN3 c.569dupG.
The work funded in the Weimer lab is centered on identifying novel therapies for the treatment of CLN3-Batten disease [(including small molecules, anti-sense oligonucleotides (ASO)]. In order to do this, the team uses in vitro high content screening assays on a combination of cell types, include patient derived fibroblasts, CLN3-deficient mouse embryonic fibroblasts, and CLN3-deficient mouse cortical neurons as well as in vivo histopathological assessment. 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 wildtype CLN3, the team will work closely with Dr. Hastings to understand which protein partners (if any) are lost when CLN3 is targeted using ASOs that skip exons 5, 7, and 8 as well as define how the wildtype CLN3 protein partners vary between cell types. These exciting experiments will not only help uncover potential new therapeutic targets but will help the field better understand the basic biology of CLN3.
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.