New York, Oct. 04, 2023 (GLOBE NEWSWIRE) -- The Hereditary Disease Foundation has announced that the recipients of its first Transformative Research Awards are two teams - one led by Beverly Davidson, PhD of Children’s Hospital of Philadelphia and the University of Pennsylvania, and the other led by Ricardo Mouro Pinto, PhD of Massachusetts General Hospital and Harvard Medical School. Each team will receive funding of $1 million over the next two years for projects focused on developing treatments for Huntington’s disease. Dr. Davidson’s and Dr. Mouro Pinto’s teams each use cutting-edge methods but take different approaches to advance Huntington’s disease research that, if successful, could lead to truly transformative therapies for Huntington’s disease.
The Transformative Research Awards were made possible through a partnership between the Hereditary Disease Foundation and a generous group of anonymous donors. They are the largest grants ever awarded by HDF.
“These new awards are designed to move the most innovative work in Huntington’s disease from concept to practice,” said Meghan Donaldson, CEO of the Hereditary Disease Foundation. “We are funding collaborative research teams who are focused on creating new ways to move toward a disease-modifying treatment and provide transformational new insights to the Huntington’s disease research field. We thank our donors whose extraordinary generosity and vision have made the Transformative Research Awards possible, and we congratulate our first recipients.”
Dr. Davidson will collaborate with Leslie Thompson, PhD from the University of California, Irvine, and Jang-Ho Cha, MD, PhD from Latus Biosciences. Their project, “Translational Studies on PIAS1 and MSH3 Knockdown for Huntington’s Disease,” will target two proteins using an adeno-associated virus (AAV) that can be injected into the brain at very small volumes and achieve wide distribution throughout the brain. MSH3 is involved in a biological phenomenon called “somatic instability,” which is the molecular stutter in the CAG repeat that causes HD to expand over time in vulnerable tissues, particularly the brain. PIAS1 has been shown to significantly improve symptoms in mice that model HD.
Dr. Davidson has been working on Huntington’s disease for decades, most recently specializing in developing and improving viral delivery strategies for HD therapeutics. She also has experience moving promising drug targets to primate models. Dr. Thompson is a renowned leader in stem cell research and has extensive experience working with various models of HD mice. Dr. Cha has worked in the pharmaceutical sector for decades and is well-versed in taking drugs from conception to clinic. Both Drs. Thompson and Cha were key members of the research team who traveled to Venezuela annually on a quest to find the gene that causes Huntington’s disease.
Dr. Mouro Pinto will work with a large team of experts including James Gusella, PhD and Benjamin Kleinstiver, PhD from Massachusetts General Hospital and Harvard Medical School; David Liu, PhD and Benjamin Deverman, PhD from the Broad Institute of MIT and Harvard; Joseph Nabhan, PhD from Vesigen Therapeutics; Cathleen Lutz, PhD, MBA from the Jackson Laboratory; and Vanessa Wheeler, PhD from Massachusetts General Hospital and Harvard Medical School. Their project, “Therapeutic Targeting of Somatic CAG Expansions with Precise CRISPR Base Editing,” will develop genome editing therapeutics that target the cause of the CAG repeat expansion in Huntington’s disease. Their goal is to permanently halt somatic instability with a one-and-done (gene editing) approach to the brains of mice that model Huntington’s disease.
Dr. Mouro Pinto has been working on Huntington’s disease since his postdoctoral studies in 2015 with Dr. Wheeler as his mentor. For this project, he has put together a team that will be a tour de force. Drs. Wheeler and Mouro Pinto helped define somatic instability in HD and identified genes targeted in this project as being involved in this process. Dr. Gusella has been working on HD since the 1970s and was a key member of the team that identified the gene that causes HD. He is a distinguished geneticist who helped lead the field in large genomic studies that identified genes that modify age of symptom onset in HD. Dr. Kleinstiver is a leader in genome editing for the treatment of human diseases. Dr. Liu invented the modified CRISPR technology, called base editing, used in this proposal. Dr. Deverman is a world expert in virus development. Dr. Nabhan is the Chief Scientific Officer of Vesigen Therapeutics, a company that specializes in packaging therapeutics so they can be delivered into the brain. Dr. Lutz is the Vice President of the Rare Disease Translational Center at the Jackson Laboratory, the world leader in developing and breeding mouse models. Dr. Wheeler has dedicated her career to understanding how somatic instability affects the onset and progression of HD.
About the Hereditary Disease Foundation
The Hereditary Disease Foundation (HDF) funds innovative scientific research to cure Huntington’s disease (HD), a genetic disorder that typically strikes in mid-adulthood, destroying brain cells and causing irreversible decline in control of mood, memory, and movement. Since HD is caused by a single gene, it serves as a model to potentially unlock cures for other complex brain disorders like Parkinson’s, Alzheimer’s, and Lou Gehrig’s (ALS) diseases. Research organized by HDF led to the discovery of the genetic marker for Huntington’s disease in 1983. HDF organized and funded a decade-long international collaboration of over 100 scientists who discovered the gene that causes HD in 1993. This work played an important role in the development of the Human Genome Project.
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