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Berkeley Lab scientists help develop promising therapy for Huntington’s disease

November 1, 2012 by · Leave a Comment 

There’s new hope in the fight against Huntington’s disease. A group of researchers that includes scientists from the U.S. Department of Energy’s Lawrence Berkeley National Laboratory (Berkeley Lab) have designed a compound that suppresses symptoms of the devastating disease in mice.

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New studies hint at possible approaches to protect those at risk for Huntington’s disease

June 18, 2012 by · Leave a Comment 

In Huntington’s disease, abnormally long strands of glutamine in the huntingtin (Htt) protein, called polyglutamines, cause subtle changes in cellular functions that lead to neurodegeneration and death. Studies have shown that the activation of the heat shock response, a cellular reaction to stress, doesn’t work properly in Huntington’s disease. In their research to understand the effects of mutant Htt on the master regulator of the heat shock response, HSF1, researchers have discovered that the targets most affected by stress are not the classic HSF1 targets, but are associated with a range of other important biological functions. Their research is published in the inaugural issue of The Journal of Huntington’s Disease.

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Reach2HD, a Phase II study in Huntington’s disease, launched

June 6, 2012 by · Leave a Comment 

The Huntington Study Group (HSG), under the leadership of Ray Dorsey, M.D. with Johns Hopkins Medical and Diana Rosas, M.D. with Massachusetts General Hospital, is conducting a clinical trial in Huntington’s disease (HD) throughout the United States and Australia, “A randomized, double-blind, placebo-controlled, study to assess the safety and tolerability, and efficacy of PBT2 in patients with early to mid-stage Huntington’s disease” comparing a 100 mg dose or 250 mg dose versus placebo. The HSG is a not-for-profit group of physicians and other clinical researchers who are experienced in the care of HD patients and dedicated to clinical research of the disease. This trial is sponsored by Prana Biotechnology Limited (Melbourne, Australia) and is being managed by the University of Rochester Medical Center.

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Researchers restore neuron function to brains damaged by Huntington’s disease

May 28, 2012 by · Leave a Comment 

Researchers from South Korea, Sweden, and the United States have collaborated on a project to restore neuron function to parts of the brain damaged by Huntington’s disease (HD) by successfully transplanting HD-induced pluripotent stem cells into animal models.

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New knowledge on the pharmacology of dopamine stabilizers

February 23, 2012 by · Leave a Comment 

A study from Karolinska Institutet in Sweden shows that a new drug for Huntington’s disease – pridopidine or dopamine stabiliser ACR16 – might operate via previously unknown mechanisms of action. Researchers have found that at very low concentrations, ACR16 binds to the sigma-1 receptor, a protein in the brain important to neuronal function and survival. This new knowledge can be used to develop future treatments for schizophrenia, involuntary Parkinsonian tremors and neurodegenerative diseases.

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U of A medical researchers discover ‘very promising’ treatment for Huntington disease

February 12, 2012 by · Leave a Comment 

Medical researchers at the University of Alberta have discovered a promising new therapy for Huntington disease that restores lost motor skills and may delay or stop the progression of the disease based on lab model tests, says the lead researcher. Because the new therapy uses a molecule already being used in clinical trials for other diseases, it could be used in a clinical trial for Huntington disease within the next one to two years.

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UC Davis investigators achieve important step toward treating Huntington’s disease

January 18, 2012 by · Leave a Comment 

A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington’s disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. The findings, now available online in the journal Molecular and Cellular Neuroscience, suggest a promising approach that might block the disease from advancing.

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Regulatory enzyme overexpression may protect against neurodegeneration in Huntington’s disease

December 17, 2011 by · Leave a Comment 

Treatment that increases brain levels of an important regulatory enzyme may slow the loss of brain cells that characterizes Huntington’s disease (HD) and other neurodegenerative disorders. In a report receiving advance online publication in Nature Medicine, a Massachusetts General Hospital (MGH)-based research team reports that increased expression of Sirt1, one of a family of enzymes called sirtuins, in the brain of a mouse model of HD protected against neurodegeneration. They also identified a potential mechanism for this protective effect.

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Dantrolene protects neurons from Huntington’s disease

November 23, 2011 by · Leave a Comment 

Huntington’s disease (HD) is characterized by ongoing destruction of specific neurons within the brain. It affects a person’s ability to walk, talk, and think – leading to involuntary movement and loss of muscle co-ordination. New research published in BioMed Central’s open access journal Molecular Neurodegeneration shows that the RyanR inhibitor Dantrolene is able to reduce the severity of walking and balance problems in a mouse model of HD.

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New insight into the cellular defects in Huntington’s disease

October 9, 2011 by · Leave a Comment 

Huntington disease is a devastating neurogenerative disorder that causes a progressive loss of functional capacity and reduced life span. It is an inherited condition caused by a mutant HTT gene. Although this has been known for many years, the functions of the normal Htt protein and the mechanisms by which the mutant protein generated from the mutant HTT gene causes disease are not well understood. A team of researchers led by Frédéric Saudou, at the Institut Curie, France, has now uncovered a new function for normal Htt protein and determined that this function is disrupted in a mouse model of Huntington disease and in patients with the disorder.

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