The Hereditary Disease Foundation (HDF) is pleased to announce the recipient of our $1 million 2024 Transformative Research Award. Dr. Andrew Yoo from Washington University School of Medicine in St. Louis and Dr. Osama Al Dalahmah from Columbia University Irving Medical Center will partner on this 2-year grant to advance research on two novel molecules that have potential as therapeutic targets for Huntington’s disease (HD).
Dr. Yoo and Dr. Osama’s project, “Uncovering Huntington’s Disease-Associated Human-Specific Non-Coding Transcripts as Therapeutic Targets,” will deeply examine two non-coding RNAs: HD-LINC and HINT, to determine their therapeutic potential in treating HD. Non-coding RNAs are pieces of genetic material that the cell never intends to turn into proteins, but they themselves perform regulatory functions throughout the cell. This innovative approach to identify targets that are specifically expressed in humans suggests the presence of these molecules is related to the disease itself and not just the genetic presence of the HD gene. Ultimately, this proposal will provide go/no-go decision criteria on advancing these two targets that represent a novel class of therapeutics for the HD space.
“We are thrilled to present this Award to Drs. Yoo and Al Dalahmah, both of whom have demonstrated a profound commitment to Huntington’s disease research and have formed a unique partnership that capitalizes on each of their specific areas of expertise,” says Meghan Donaldson, Chief Executive Officer of the HDF. “This is the third Transformative Research Award presented by the Hereditary Disease Foundation and we are confident that this award mechanism will continue to promote truly novel and exceptional research and encourage cooperation among scientists working toward treatments for Huntington’s disease.”
Andrew Yoo (principal investigator), Osama Al Dalahmah (co-investigator)
Dr. Yoo is a world-leader in stem cell research and has developed a direct conversion protocol that turns skin cells directly into brain cells. This protocol is used by labs across the globe that study HD. Directly converting skin cells to brain cells preserves cellular age along with other disease features disguised by youth. Such a system pulls out degenerative features of the cells, making it a more robust system with which to study diseases like HD that are brought on by age.
“We’re grateful for the opportunity to collaborate on this project. We hope that the mechanisms we define will reveal pathways that can be targeted for potential therapeutic interventions in the future,” says Dr. Yoo.
Dr. Al Dalahmah is a neuropathologist and a renowned expert in brain structures and the effects of HD within the central nervous system. Trained under the tutelage of Dr. John Paul Vonsattel, the creator of the grading system that has been used for over 40 years to categorize HD brains, Dr. Al Dalahmah is a talented researcher focused on dissecting the molecular effects of HD with direct access to the New York Brain Bank at Columbia University, one of the largest repositories in the world of human HD brains.
“The drive to find a cure for Huntington’s disease has brought the Yoo and Al Dalahmah groups together. With the support of this Transformative Research Award, we will collaboratively explore innovative, out-of-the-box ideas and targets based on findings from human HD, bringing us closer to a clinical cure,” says Dr. Al Dalahmah.
“Research that identifies HD-related changes that specifically happen in humans is what we need for advancing drugs that will work in people,” says Sarah Hernandez, PhD, Director of Research Programs for the HDF. “It’s thrilling to see work like this that is advancing novel targets. Moving various projects forward that take totally different approaches ensures that we’re targeting HD from various angles. I’m eager to see what Drs. Yoo and Al Dalahmah find. Congratulations to the team!”
The Transformative Research Awards were made possible through a partnership between the HDF and a generous group of anonymous donors. They are the largest grants ever awarded by the Foundation.