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September 17, 2019September 17, 2019

Age of Onset in Huntington’s Linked to Property of CAG Repeat Length in Study

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Age of onset in Huntington’s disease is associated with a property of the inherited CAG repeat length in the huntingtin (HTT) gene — which determines the probability of further repeat ... Read more

September 10, 2019September 10, 2019

New Small Molecule AZ67 May Help Prevent Brain Cell Death, Early Data Show

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A small molecule that alters glucose metabolism in stressed brain cells may help prevent their death, representing a new strategy for treating brain diseases including amyotrophic lateral sclerosis, Alzheimer’s ... Read more

September 5, 2019September 5, 2019

New Gene-editing Tool SATI Can Target Different Kinds of Disease-causing Mutations, Mouse Study Suggests

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A new gene editing method allows researchers to target many different kinds of disease-causing mutations in multiple cell types, which could have implications for genetic disorders such as Huntington’s ... Read more

September 3, 2019September 3, 2019

Subtle Changes in Speech Mark Pre-symptomatic Huntington’s, Study Finds

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People at a pre-symptomatic, or premanifest, stage of Huntington’s disease (HD) have subtle speech alterations that may be used as a marker to evaluate new therapies intended to stop ... Read more

August 30, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 29, 2019

Study Links Sensitivity to Loss with Apathy In Huntington’s Patients

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Being less affected by loss is predictive of apathy in people with Huntington’s disease (HD), a new study has found. The study, “Insensitivity to Loss Predicts Apathy in Huntington’s Disease,” ... Read more

August 29, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 27, 2019August 27, 2019

Tiny Lipids as Delivery Vehicle May Make CRISPR-Cas9 More Effective Gene Editing Tool

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Tiny fatty (lipid) particles can be used to enhance the delivery of gene editing tools, such as CRISPR-Cas9, to targeted cells and considerably improve their ability to possibly treat ... Read more

August 20, 2019August 20, 2019

Researchers Find Genetic Overlap Between Psychiatric Disorders and Psychiatric, Cognitive Symptoms in Huntington’s

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Psychiatric and cognitive symptoms in Huntington’s disease share genetic traits with psychiatric disorders and intelligence, respectively, according to a study. These findings suggest that current treatments used for depression ... Read more

August 20, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

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