Mice Research Offers Possible Approach to Stopping Huntington's Disease

Researchers at the University of Iowa in Iowa City this month made public the results of their research into stopping dominant progressives brain disorders in mice.

A dominant progressive brain disorder is a disease caused when an infant inherits one copy of a mutant gene which results in the production of mutant proteins that cause loss of cognitive abilities. The most widely occurring form of this sort of disease in the United States is Huntington’s disease, with as many as 250,000 sufferers in this country alone. Huntington’s disease usually manifests itself in a victim’s mid-to-late 30s and sufferers typically live only 15-20 years after diagnosis while suffering progressively worsening brain degeneration.

The University of Iowa researchers used gene therapy to treat mice afflicted spioncerebellar ataxia type 1. The disease produces a mutated protein in the brain that eventually leads the mice to have difficulty in walking. Like Huntington’s it is not a result of a missing gene — which gene therapy has traditionally been used to treat in animal models and in a small number of humans — but rather a mutant gene inherited from a parent.

Nonetheless, researchers used a virus to carry modified genetic material to the mice. The genetic material was designed to bind with and block the expression the defective gene. After the mice were injected with the virus, the production of the proteins causing the disease stopped and the mice appeared to improve their ability to walk.

The researchers then took the process one step further and used the same method to see if genetic material could be effective when added in vitro to human Huntington’s cell cultures. After exposing the cell cultures to a different virus containing the genetic materials, the cells stopped producing the proteins that cause Huntington’s disease.

Lead researcher Beverley Davidson said the she hopes this discovery moves quickly into clinical trials to see if it can be effective in treating Huntington’s in human beings,

The data are very promising; we hope we will be able to use RNA interference as a therapy for dominant neurodegenerative diseases.


Cure hoped for Huntington’s sufferers. Erika Chek, Nature, June 9, 2004.

2 thoughts on “Mice Research Offers Possible Approach to Stopping Huntington's Disease”

  1. Animals aren’t human.
    Why don’t we use mass murderers and serial rapists to test these drugs on them and maybe we would have found a cure faster.

    At least animals has never done anything wrong as these horrible human beings did a lot.

    We make new stuff and later we find out they cause cancer, they’re bad for us in long terms. Because animals aren’t people and maybe if we test these things on humans with negative values such as mass shooters, rapists, murderers and other horrible people on deathrow we would know what is good and what is bad.

    Stop animal testing – it’s not just cruel, it’s ineffective
    By Kelly Overton
    June 23, 2006 The pharmaceutical industry and the National Institutes of Health spend billions of dollars annually on medical research techniques that have been rendered obsolete by technological advances.

    Adult stem cell research is key to our status as the world’s leader in medical research. The continued use of animals to test the effectiveness of medications and health interventions for humans is akin to using smoke signals instead of e-mail as a method of communication.

    Animal testing has never really worked. Animal tests proved penicillin deadly, strychnine safe and aspirin dangerous.

    In fact, 90 percent of medications approved for human use after animal testing later proved ineffective or harmful to humans in clinical trials. It is humbling to realize that the flipping of a coin would have proved five times more accurate and much cheaper. Animal-tested drugs have killed, disabled or harmed millions of people and lead to costly delays as well. Among the most publicized are the delays of a polio vaccine by over three decades and a four-year delay in the use of protease inhibitors for HIV treatment – after animal testing showed these interventions to be useless.

    We have spent billions of dollars to cure cancer in mice, but so far have failed to replicate human cancer in any animal, let alone close in on a cure. All but a very few diseases are species-unique, and the only efficient and effective way to discover cures and create vaccines is through the use of the same species’ cells, tissues and organs.

    The use of animals as models for the development of human medications and disease almost always fails, simply because humans and animals have different physiologies.

    Adult stem cell research is more effective than animal testing because there are no complications or failures related to tissue rejection. In fact, international researchers using adult stem cells – cells that are present in all growing human tissue – have shown success in treating cardiac infarction, Crohn’s disease and thalassemia. The answers to the mysteries of Parkinson’s and Alzheimer’s will be found by using stem cells and other modern technologies, not by cutting up beagles.

    Most Americans tolerate vivisection because they believe that it is a necessary evil. It is evil, but it’s not necessary. Whether vivisection is morally right or wrong no longer matters: It is as obsolete as eight-track tapes, telegrams and bloodletting. It is time the public stopped funding this antiquated science, through tax dollars and research and development costs imbedded in prescription prices.

    It may even be time to consider lawsuits aimed at pharmaceutical companies that continue to profit by charging patients, insurance companies and the state and federal governments for medications and treatments based on such flawed and antiquated research. These lawsuits could rival the tobacco lawsuits of the past decade, with individuals and states seeking damages for the cost of caring for those killed or disabled by dangerous medicines.

    Regardless of one’s feelings about animals, it is time for consumers and taxpayers to realize that vivisection wastes hundreds of millions of dollars annually and produces an inferior product.

    The medical progress of the past century is the result of technology, public health improvements, epidemiology, human clinical research, human autopsies, mathematical modeling and the mapping of the human genome, not experiments on animals.

    The NIH must take responsibility for ensuring the United States maintains its status as the world’s leader in health care innovation, a position that guarantees our country’s future economic strength and protects the world from the growing threat of biological terrorism. This responsibility begins by ensuring that the research funded with Americans’ tax dollars uses the most modern technology and methodology.

    Whether you will live a full life or die early probably depends on today’s medical research. Researchers have proved ad infinitum that hitting a beagle on the head with a hammer causes trauma and forcing monkeys to smoke gives them cancer.

    It’s time to insist that they stop harming defenseless animals and wasting our precious health care dollars so they can get busy saving our lives by embracing technologies that work.

    The site accepts fair use which is NOT for commercial use. My site is a non commercial and non-profit website.
    Six Reasons why Animal Testing Doesn’t Work

    Human and animal testing agree only 5-25% of the time, according to Huntingdon Life Sciences
    88% of stillbirths are due to drugs posed to be safe in animal testing
    According to World Health Organization out of 200,000 released mediations only 240 are labeled as essential
    Corneal transplants were delayed for 90 years and blood transfusions were delayed 200 years due to animal studies
    Animal experiments can be replaced by at least 450 methods known at this time
    Less then 2% of human illnesses or 1.16% are ever seen in animals

    Ninety-four percent of animal testing is done to determine the safety of cosmetics and household products leaving only 6% for medical research! Cosmetic testing is banned in Belgium, Netherlands and the U.K.. Europe has been phasing out all products related to animal testing since 2002 and they plan to completely ban all products by 2009. This is a big step in right direction for millions of animals who were helplessly killed during tests for cosmetics and household products. Unfortunately the U.S. is still home to many companies who continue to legally perform horrible test on animals even though the United States Food and Drug Administration (FDA) and the Consumer Product Safety Commission doesn’t require animal testing for cosmetics or household products!
    Most of the animals that are used in testing are bred just for testing, but many others come from the pound. Mice, rabbits, dogs, guinea pigs, cats and monkey’s are the most commonly used animals for tests. It has been proven that there is already enough existing safety data, as well as in vitro (test tube) alternatives to make animal testing for cosmetics and household products even more unnecessary and unethical. By just listing the names of the tests I will be able to give you a better idea of what these poor animals go through. Whole Body, Short-term Toxicity, Skin Penetration, Skin Irritancy, Eye irritancy, Skin Sensitization, Phototoxicity & Photosensitisation, Mutagenicity, Carcinogenicity, Reproductive Toxicity, Teratogenicity and Finished Product Testing are all common tests performed on animals.

    The LD50 test short for lethal dose, is one of the worst tests that was developed back in 1927 and is still in use today. Groups of animals are dosed with different amounts of a test substance in order to determine the dose which kills half of the animals! Animals are often force-fed the substance. The LD50 test is known to use huge, unrealistic doses that are completely unrelated to possible exposure levels. There are now other tests available that use less animals and lower doses, yet this old, discredited LD50 test continues. During another common test, the Draize eye-and skin-irritation test, rabbits are immobilized in full-body restraints while a substance is dripped or smeared into their eyes or onto their shaved skin. Rabbits often scream in pain and many break their necks trying to get free. The Draize test has been proven in studies to “grossly over predicted the effects that could be seen in the human eye, and does not reflect the eye irritation hazard for man”. The human four-hour patch skin test has proved to provide chemical skin-irritation data that are “inherently superior to that given by a surrogate model, such as the rabbit.”

    With so much going on in the world and our lives, I know it easy to feel overwhelmed and helpless. But by reading this article you have proved that you do care. I know it is hard and horrible to even think of what is happening behind closed doors. But right now there are millions of animals who are caged, tortured, and can’t speak for themselves. You can make progress and reach out to help them by taking just a second to sign this online petition here. By not purchasing products from companies that continue to test on animals you are also sending the message that it is not right.

    Animal testing – Dangerous to human health
    Overwhelming evidence demonstrates that animal tests are dangerous to human health, and may be the reason that so many ‘safety tested’ drugs cause so many side effects.

    Animal testing doesn’t work.

    Its results are often inconclusive and cannot be accurately extrapolated to humans. As a result, relying on the results of animal testing can be dangerous to human health. It is a system which is long overdue for a critical review, and yet no such review is on the horizon.

    In his seminal book, the Naked Empress: The Great Medical Fraud (Switzerland: CIVIS, 1992) eminent researcher Hans Ruesch notes that approximately 15,000 new drugs are marketed every year, while some 12,000 are withdrawn. According to the Food and Drug Administration (FDA), 1.5 million Americans were hospitalised in 1978 alone as a consequence of pharmaceutical drugs administered to “cure” them. A further 30 per cent of all hospitalised people suffered further damage from the therapy prescribed to them. In the 1990s, studies…

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