All posts by Alzforum, Tom Fagan

First Phase 2 Success for Gene Therapy in Parkinson’s

Injecting millions of viral particles deep into the brain may sound nefarious, but when the particles are relatively benign and carry the gene for the enzyme glutamic acid decarboxylase (GAD), then the strategy may have the makings of a therapy for Parkinson’s disease (PD). As reported in the March 17 Lancet Neurology online, PD patients […]

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WEBINAR: Stress Granules and Neurodegenerative Disease—What’s the Scoop?

The formation of cytoplasmic inclusions called stress granules is one of nature’s many ways of coping with adverse situations. It appears that these inclusions sequester non-essential messenger RNAs, allowing cells to focus on making proteins, such as chaperones, that protect against a variety of stressful insults. Recent work suggests that stress granules may figure in […]

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New Strategy Nets Biomarkers for AD, and More

It met with little enthusiasm when originally proposed, and took more than three years to perfect, but a new strategy for detecting serum antibodies could have important ramifications for Alzheimer’s disease diagnostics. Like many groundbreaking ideas, Thomas Kodadek’s was profoundly simple—use a huge library of synthetic antigens to capture circulating antibodies that are specific to […]

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Sorting Progranulin With Sortilin—New Clues to FTLD Pathology

Progranulin, a raw form of granulin (GRN), landed center stage of neurodegeneration research four years ago (see ARF related news story), when scientists discovered that loss-of-function mutations in the GRN gene cause a specific form of dementia—frontotemporal lobar degeneration with ubiquitin inclusions (FTLD-U). Fascination with the protein grew when studies later identified GRN missense mutations […]

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Iron Export? New Role Links APP, Metals, to Oxidative Stress

No, you haven’t accidentally hit the home page of the World Trade Organization. Amyloid precursor protein (APP) does export iron—from neurons—according to a paper in the September 2 Cell online. Researchers led by Ashley Bush at the University of Melbourne, Victoria, Australia, and Jack Rogers at Harvard Medical School report that APP oxidizes Fe2+ to […]

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The Ubiquitin Proteasome System—Out of STEP in AD?

30 April 2010. You might be inclined to wonder about your refuse collector if garbage was piling up in your neighborhood. What about in your brain? For years scientist have looked to cellular recycling mechanisms to explain the accrual of toxic protein aggregates, such as the senile plaques and neurofibrillary tangles found in Alzheimer disease, […]

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BDNF—Learning Boosts Trophin Signals in Hippocampus

Use it or lose it has become a self-help mantra for people trying to beat the odds of getting Alzheimer disease (AD). But exactly how does using one’s brain defend against the dreaded loss of faculties One hint comes in a soon-to-be-published paper in PNAS online. Researchers led by Christine Gall at the University of […]

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Dimebon Disappoints in Phase 3 Trial

4 March 2010. One swallow does not make a summer, and one positive clinical trial does not make an Alzheimer’s drug. In 2007, expectations for Dimebon, an old antihistamine, soared on results of a positive Phase 2 trial in AD patients in Russia. Dimebon appeared not only to slow cognitive decline, but even to improve […]

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Baby Steps for Gene Therapy? Fatal Infant Disease Cured in Mice

Is anyone, parent or physician, brave enough to allow gene therapy in newborn babies The answer may come sooner than we think. In Sunday’s Nature Biotechnology online, researchers led by Brian Kaspar at Nationwide Children’s Hospital, Columbus, Ohio, report remarkable success in rescuing spinal muscular atrophy (SMA) in mice. The rescue came after injecting one-day-old […]

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