HD News Site. Gene Therapy News. Gene therapy could correct spelling mistakes in our DNA, while stem cells promise transplant operations that don’t need powerful immune suppressing drugs. Scientists have now combined the two techniques in a genetic liver disease. Several challenges remain before this could work in Huntington’s disease though. Huntington’s disease is one of many conditions caused by a genetic error. A mistake in the genetic recipe for the huntingtin protein causes HD. All cells carry this mistake in their genetic make-up and, as a result, produce the abnormal protein. Common sense suggests the possibility of just going into cells and ‘surgically’ removing the faulty bit. But thinking about how large the entire genome is, in relation to the small size of the HD mutation - 6 billion letters versus just a few dozen extra letters in HD - recalls a search for a needle in a haystack. Stem cells can divide into new stem cells, and develop into specialized cells including neurons.
Stem Cell News. Many people consider gene silencing the best candidate therapy for Huntington’s disease. A group led by Dr Jan Nolta has described a new method of getting silencing drugs into cells in a dish using modified bone marrow stem cells. Does this new approach offer benefits over existing technologies? Like most HD researchers, we think gene silencing, or shutting off the expression of the HD gene, is the most exciting treatment option on the short term horizon.
To achieve this goal, drugs that block expression of the HD gene must be delivered to the brain in some way. The problem is twofold: we must first develop drugs that silence the HD gene, and then get the to the brain. For good reasons, your body goes to great lengths to protect your brain. When we zoom in and look closer, we realize that the brain is even better protected than it appears from the outside. So far, gene silencing studies have used one of two methods to deliver drugs to the brain. A group of scientists led by Dr. HD Extra Simplified. More General Information. Potential Stem Cell Treatment. Public release date: 15-Mar-2012 [ Print | E-mail Share ] [ Close Window ] Contact: Su-Chun Zhang zhang@waisman.wisc.edu 608-265-2543University of Wisconsin-Madison MADISON -- Huntington's disease, the debilitating congenital neurological disorder that progressively robs patients of muscle coordination and cognitive ability, is a condition without effective treatment, a slow death sentence.
But if researchers can build on new research reported this week (March 15, 2012) in the journal Cell Stem Cell, a special type of brain cell forged from stem cells could help restore the muscle coordination deficits that cause the uncontrollable spasms characteristic of the disease. "This is really something unexpected," says Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the new study, which showed that locomotion could be restored in mice with a Huntington's-like condition.
The new study was funded by the U.S. Terry Devitt 608-262-8282, trdevitt@wic.edu. HD Society of America. Stem Cell Info. Stanford Research / Outreach. “Alive and Well” Screening at Roble Hall Tuesday, March 4th 7-9pm at Roble Theater Free Parking Available Outside of Roble Hall (374 Santa Teresa St) ***** Welcome New Visitors! Start here! The HOPES website is currently undergoing some design changes. All of our content is still here, so feel free to use the search bar at the top or navigate our menus. Here are links to our popular articles by category. HD Basics – HD in a nutshell, Symptoms, Juvenile HDHD GeneticsHD and the Brain – HOPES Brain Tutorial, Neurobiology, Other neurodegenerative disordersDrugs and Supplements – Alphabetical Listing, Listing by Function, HD ScorecardResearch and HD – Research Basics, Research Updates, HD Scientists, Research InstitutionsManaging HD – HD Advocacy, Lifestyle and HD, Diet and HDFor KidsOther Resources – HOPES News, Helpful Links, Literature Corner ***** Please let us know your thoughts or any suggestions for improvement as we finalize our new design.
Genetic Testing. Individuals at risk for Huntington’s disease (HD) have the option of undergoing genetic testing, which detects the presence or absence of the genetic sequence that causes HD. The decision of whether or not to undergo genetic testing is intensely personal, with many factors to consider. This chapter will provide scientific background information regarding genetic testing for Huntington’s disease. (For more information on how testing for HD may affect relationships, click here ) How is genetic testing for HD possible? In 1983, a team of scientists located the first genetic marker for Huntington’s disease, indicating the approximate location of the Huntington gene on chromosome 4 . In 1993, the Huntington’s Disease Collaborative Research Group isolated the Huntington gene and identified the hereditary version of this gene that causes HD.
Are there guidelines for genetic testing? Who is at risk for HD? When is an individual tested for HD? Where does genetic testing occur? Hersch S, et al. HD Future. In order to make predictions about the future spread or decline of Huntington’s Disease, we can draw upon scientific tools from the field of population genetics (see Part I for an introduction to this field). According to population genetics, genes that make it more difficult for an affected individual to survive to reproductive age will tend to decrease in frequency in a population over time. Such a decrease in frequency is an example of the more general process of natural selection—the elimination of variation that results from differential survival and reproduction.
The gene for HD is a special case in that it primarily affects men and women once they have passed their reproductive years. As a result, those affected by HD as adults will already have reproduced and, by the normal laws of probability (as described here), may already have passed on the harmful gene to their children. So if natural selection plays little role, what other evolutionary forces act on Huntington’s Disease? Stem Cell Treatment News. Washington: A new technique has been developed by researchers which uses stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington’s disease. Huntington’s is a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. This new approach might block Huntington’s from advancing. Researchers think that the best chance to halt the disease’s progression will be to reduce or eliminate the mutant huntingtin (htt) protein found in the neurons of those with the disease.
“For the first time, we have been able to successfully deliver inhibitory RNA sequences from stem cells directly into neurons, significantly decreasing the synthesis of the abnormal huntingtin protein,” Jan A Nolta, principal investigator of the study and director of the UC Davis stem cell program and the UC Davis Institute for Regenerative Cures, was quoted as saying.
Huntington's Disease Statistics. Huntington's Disease Statistics: An Introduction Huntington's disease (also known as Huntington disease or HD) is a progressive brain disorder that causes uncontrolled movements, cognitive difficulties, and emotional disturbances. Huntington's Disease Statistics Regarding Prevalence Huntington's disease affects an estimated 3 to 7 per 100,000 people of European ancestry. The disorder appears to be less common in some other populations, including African Americans and Japanese. In the United States alone, about 30,000 people have Huntington's disease; estimates of its prevalence are about 1 in every 10,000 people. At least 150,000 others have a 50 percent risk of developing Huntington's disease, and thousands more of their relatives live with the possibility that they, too, might develop Huntington's disease.
Huntington's Disease Statistics Concerning Inheritance Risk Each parent has two copies of every chromosome but gives only one copy to each child. HD General Information. For more information on neurological disorders or research programs funded by the National Institute of Neurological Disorders and Stroke, contact the Institute's Brain Resources and Information Network (BRAIN) at: akinesia-decreased body movements. at-risk -a description of a person whose mother or father has HD or has inherited the HD gene and who therefore has a 50-50 chance of inheriting the disorder. autosomal dominant disorder -a non-sex-linked disorder that can be inherited even if only one parent passes on the defective gene. basal ganglia -a region located at the base of the brain composed of four clusters of neurons, or nerve cells.
This area is responsible for body movement and coordination. Caudate nuclei -part of the striatum in the basal ganglia. Chorea -uncontrolled body movements. Chromosomes -the structures in cells that contain genes. Computed tomography (CT)- a technique used for diagnosing brain disorders. Kindred -a group of related persons, such as a family or clan. Research Advance in Treatment.
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