On 6/29/22, the HOPE for Harvey Foundation signed a contract with Everlum Bio, a rare disease lab who believes that everyone deserves a cure, no matter how big or small the disease. Everlum Bio is based in Austin, TX, and we are excited to partner with them on creating a treatment for DLG4-related Synaptopathy, focusing on ASOs and small molecule screening.
On 3/16/23, HOPE for Harvey signed an additional contract with Hebrew University of Jerusalem and Rami Aqeilan's lab in collaboration with the SHINE Foundation to support the research and development of a virus-based gene replacement therapy called an AAV.
In the summer of 2023, the HOPE for Harvey Foundation applied for two DLG4 animal model grants at Jackson Laboratories (Jax) and the Center for Precision Animal Modeling (C-PAM) at the University of Alabama at Birmingham (UAB), and both were accepted and are currently in progress.
We are currently working on these specific pre-clinical development tasks with funds from this GoFundMe/GiveButter Campaigns and HOPE for Harvey Foundation Fundraising Efforts:
Check-out this video from our AAV project with Rami Aqeilan, Hebrew University of Jerusalem
Currently there is no treatment options for DLG4-related Synaptopathy. We hope to change that with your help! 100% of donations will support the development of various treatment modalities.
Due to advocacy by HOPE for Harvey Foundation, four additional academic labs recently started research projects on DLG4-related Synaptopathy:
"Collins concluded by thanking his audience while urging them to do more. 'Of nearly 7,000 genetic disorders, only 500–600 have an FDA-approved therapy,' he said. The goal is to develop therapies for the thousands of disorders for which patients and their families are still waiting in vain."
"After excluding genetic variants that were also found in the control group and in a separate large cohort of more than 60,000 individuals without autism, Doan, Yu and colleagues were left with 41 genes that were knocked out only in individuals with autism. Overall, the researchers estimate that these genes explain another 3 to 5 percent of all cases of autism (2 percent from loss-of-function mutations, and 1 to 3 percent from missense mutations)."
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