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Scientists Claim Encouraging Progress On Hereditary Diseases

NA. The RNA then makes a protein, which is also abnormal. This abnormal protein wreaks havoc and eventually is the cause of cellular disability and death.

Extraordinary new techniques target the RNA and prevent it from ever making the abnormal protein at all. This strategy is virtually a cure not only for Huntington's disease but also for hereditary forms of Alzheimer's disease, Parkinson's disease, Lou Gehrig's disease, other genetic diseases and even cancer. The meeting presented fabulous advances in the strategies, which are called RNA interference. All of these breakthroughs are currently curing Huntington's disease in mice. They are also being tested for safety in monkeys. Participants grappled with the challenges of rapidly moving these discoveries to people.

Participants also heard the very promising results of cell transplant therapy. People with Huntington's disease in France received cellular transplants into their brains, which then grew and made connections. The initial pilot phases of this research were so encouraging, even after a follow-up of 8 years, which the research has expanded to include 60 or more HD patients in Europe. These clinical trials are being conducted in such a rigorous fashion that they will enable us to learn a great deal about the future of cell replacement therapy.

Participants also learned about significant advances in the development of new stem cell lines. These lines of neurons - or brain cells - could also benefit patients and could potentially be used as a reliable and renewable source of tissue for cell replacement therapy. Interesting and promising pilot research findings were presented using high dose creatine as a therapy for HD patients. At the mouse level, new intriguing findings were reported concerning BDNF, brain-derived neurotrophic factor. BDNF is a kind of support and survival factor without which brain cells can die - like a kind of food for brain cells. When B
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