Home  |  Top News  |  Most Popular  |  Video  |  Multimedia  |  News Feeds  |  Feedback
  Medicine  |  Nature & Earth  |  Biology  |  Technology & Engineering  |  Space & Planetary  |  Psychology  |  Physics & Chemistry  |  Economics  |  Archaeology
Top > Medicine, Health Care > Genome-scale Study Identifies Hundreds of… >

Genome-scale Study Identifies Hundreds of Potential Drug Targets for Huntington's Disease

Published: November 30, 2012.
Released by Buck Institute for Age Research  

Scientists searching for ways to develop treatments for Huntington's disease (HD) just got a roadmap that could dramatically speed their discovery process. Researchers at the Buck Institute have used RNA interference (RNAi) technology to identify hundreds of "druggable" molecular targets linked to the toxicity associated with the devastating, ultimately fatal disease. The results from this unprecedented genome-scale screen in a human cell model of HD are published in the November 29, 2012 edition of PLOS Genetics. The work was is a collaboration between Buck Institute faculty members Robert E. Hughes, Ph.D., Sean Mooney, Ph.D., Lisa Ellerby, Ph.D. and Juan Botas, Ph.D. at the Baylor College of Medicine.

HD is a devastating and incurable progressive neurodegenerative genetic disorder that affects motor coordination and leads to severe physical and cognitive decline. Currently, there are about 30,000 people in North America diagnosed with HD and another 150,000 people at risk for developing the disease. The disease pathology stems from a mutation in the huntingtin gene (HTT), resulting in the accumulation of a toxic protein leading to neuronal cell death and systemic dysfunction. Buck Scientists screened more than 7,800 genes pre-selected as potential drug targets to identify modifiers of HD toxicity in human cells, using technology that silences specific genes prior to analysis.

Lead author Robert Hughes said that among the diverse range of modifiers identified, this study showed that RRAS, a gene involved in cell motility and neuronal development, is a potent modulator of HD toxicity in multiple HD models. "Our data indicates that the pathogenic effects of the HTT mutation on this pathway can be corrected at multiple intervention points and that pharmacological manipulation of RRAS signaling may confer therapeutic benefit in HD," Hughes said. Follow up work on the RRAS pathway is now underway in the Hughes lab and in the lab of Buck faculty member Lisa M. Ellerby, PhD.

Hughes said many molecular hits identified in the screening were validated in human cell, mouse cell and fruit fly models of HD – and that all the data from the study will be available to the public. "Our hope is that HD researchers will look at these targets and find modifiers relevant to the areas they already work on," said Hughes. "Ideally, pharmaceutical companies already working on some these pathways could build on their current knowledge and expertise by focusing their attention on the challenge to develop therapies for HD."




The above story is based on materials provided by Buck Institute for Age Research.

Translate this page: Chinese French German Italian Japanese Korean Portuguese Russian Spanish


comments powered by Disqus


Related »

Response 
9/12/14 

Corn Spots: Study Finds Important Genes in Defense Response
Yield 
3/28/10 
Single Gene Dramatically Boosts Yield, Sweetness in Tomato Hybrids, CSHL-Israeli Study Finds
Cold Spring Harbor, N.Y. – Giving tomato breeders and ketchup fans something to cheer about, a Cold Spring Harbor …
Skin 
10/18/12 

A*Star Scientists Identify Mutation That Causes Skin Hyperproliferation
Underlie 
5/14/12 
Scientists Make Groundbreaking Discovery of Mutation-causing Genetic Disorder in Humans
Scientists at A*STAR's Institute of Medical Biology (IMB), in collaboration with doctors and scientists in Jordan, Turkey, Switzerland and …
Adp-Ribose 
3/12/13 
Hereditary Neurodegeneration Linked to ADP-ribose Modification
HEIDELBERG, 12 March 2013 – Attaching chains of the small molecule ADP-ribose to proteins is important for a cell's …
Single 
3/12/13 
Sleator Lab Identifies Single Point Mutation in Listeria Monocytogenes
The bacterial foodborne pathogen, Listeria monocytogenes is the causative agent of listeriosis—a debilitating disease linked with ~2,500 illnesses and …
Disease 
3/12/15 
Preventing Heart Cells from Turning to Bone
Researchers from the Gladstone Institutes have used human cells to discover how blood flow in the heart protects against …
More » 
 
© Newsline Foundation  |  About  |  Privacy Policy  |  Feedback  |  Mobile