Project will study gene regulatory network of genetic neurodegenerative disorder
Huntsville, Ala. — HudsonAlpha Institute for Biotechnology and CHDI Foundation have announced a research collaboration to study the gene regulatory network controlling the expression of the huntingtin (HTT) gene that, when mutated, causes Huntington disease, a genetic neurodegenerative disorder characterized by the progressive breakdown of nerve cells in the brain. Richard M. Myers, PhD, president and science director at HudsonAlpha is leading the project along with senior scientist Brian Roberts.
“While we know that Huntington disease is caused by a mutation in a single inherited gene, we want to learn more about the molecular network that controls the way instructions in the huntingtin gene affect specific cell functions,” Myers said.
More than 30,000 Americans suffer from Huntington disease, and another 200,000 are at risk for inheriting the disease. Every child who has a parent with Huntington disease has a 50/50 chance of also inheriting the disease gene, and everyone who inherits the disease gene develops the fatal disorder. While early symptoms include personality changes, forgetfulness, unsteady gait and slurred speech, over time, the disease affects the ability to reason, walk and speak. Ultimately, patients succumb to pneumonia, heart failure or other complications.
“A thorough understanding of the molecular network that regulates the huntingtin gene could eventually lead to therapeutic interventions for individuals suffering from the disorder,” Myers said. “And ultimately we’re optimistic that we can help untangle the genetic pathways that cause this devastating disease.”
CHDI Foundation, which is funding the collaboration, is a privately-funded, nonprofit biomedical research organization devoted exclusively to Huntington disease. Its mission is to develop drugs that will slow the progression of Huntington disease and provide meaningful clinical benefit to patients as quickly as possible. To achieve this, CHDI manages a diverse portfolio of research projects through a novel virtual model that encourages scientific collaboration to more directly connect academic research, drug discovery and clinical development.
“In collaboration with HudsonAlpha and Dr. Richard M. Myers, a leader in deciphering the regulation of human genes in health and disease, we are looking to systematically dissect the multiple mechanisms that converge to regulate the huntingtin gene,” said Sung-Wook Jang, PhD, a science director at CHDI Management. “This will have particular relevance for therapeutic strategies aimed at lowering huntingtin that are being developed for Huntington disease.”
About HudsonAlpha: HudsonAlpha Institute for Biotechnology is a nonprofit institute dedicated to innovating in the field of genomic technology and sciences across a spectrum of biological challenges. Opened in 2008, its mission is four-fold: sparking scientific discoveries that can impact human health and well-being; bringing genomic medicine into clinical care; fostering life sciences entrepreneurship and business growth; and encouraging the creation of a genomics-literate workforce and society. The HudsonAlpha biotechnology campus consists of 152 acres nestled within Cummings Research Park, the nation’s second largest research park. Designed to be a hothouse of biotech economic development, HudsonAlpha’s state-of-the-art facilities co-locate nonprofit scientific researchers with entrepreneurs and educators. The relationships formed on the HudsonAlpha campus encourage collaborations that produce advances in medicine and agriculture. HudsonAlpha has become a national and international leader in genetics and genomics research and biotech education, and includes more than 30 diverse biotech companies on campus. To learn more about HudsonAlpha, visit hudsonalpha.org.
About CHDI Foundation, Inc: CHDI Foundation, Inc. is a privately-funded nonprofit biomedical research organization that is exclusively dedicated to rapidly developing therapies that slow the progression of Huntington’s disease. Our scientists work closely with a network of more than 700 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that our common goals remain in focus. This helps bridge the translational gap that often exists between academic and industrial research pursuits and that adds costly delays to therapy development. In its role as a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as rapidly as possible. Our activities extend from exploratory biology to the identification and validation of therapeutic targets, and from drug discovery and development to clinical studies and trials. More information about CHDI can be found at www.chdifoundation.org.