Two For One? ASOs for Ataxin Allay ALS and SCA2 in Mice
Using torpedo-like DNA snippets to delete mRNAs from cells, scientists lowered the amount of ataxin-2 protein. This allayed disease in mouse models of two neurodegenerative disorders.
6390 RESULTS
Sort By:
Using torpedo-like DNA snippets to delete mRNAs from cells, scientists lowered the amount of ataxin-2 protein. This allayed disease in mouse models of two neurodegenerative disorders.
Congress has opposed the Trump administration’s request to cut NIH funding, agreeing on a $2 billion boost, with a $400 million bonus for Alzheimer’s research.
People with ALS—especially those who progress fast—express myriad inflammatory genes in their blood monocytes.
Found in familial and sporadic cases of ALS, mutations in the annexin A11 gene lead to elaborate protein clumps and seem to disrupt vesicular trafficking.
A trial of an α-synuclein antibody is making use of the technology to follow subtle changes in the day-to-day function of participants.
Even without this important sidekick at hand, scientists are forging ahead with efficacy trials of their first attempts at disease-modification in Parkinson’s
The drug slows functional decline in patients with mild disease and should be available to U.S. patients in August.
A broadening drug development pipeline now contains Phase 1 or 2 drugs on targets unrelated to Aβ or tau.
Preparing the whole mouse head—not just the dissected brain—lets researchers analyze the neural tissue outside the skull that connects the nose to the inner sanctum.
Researchers at AD/PD 2017 presented new data on an antibody and small molecules targeting amyloid.
Ionis announced top-line results of a Phase 3 trial of their drug for familial amyloid neuropathy. It met both primary endpoints but caused serious side effects in some people.
By targeting distinct cell populations in the external globus pallidus, researchers restore mobility to a mouse model of Parkinson’s disease.
Tyrosine kinase inhibitors kept diseased motor neurons alive in vitro, and notched positive findings in a recent Phase 3 trial. A new paradigm for ALS treatment?
Over the past 15 years, deaths attributable to Alzheimer’s have risen 55 percent, according to the CDC, and more people are living at home when they die, suggesting a need to support caregivers.
Protein Liquid-Liquid Phase Transitions: The Science Is About to Gel Confused about protein liquid-liquid phase transitions? You are not alone. Researchers working in this emerging field gathered in Leuven in early May to make sense of the latest data lin