TL;DR: In mice, a new type of gene-silencing molecule lowered levels of alpha-synuclein, the protein that clumps in Parkinson’s. Mice given it kept more dopamine-making brain cells and did better on grip and balance tests than untreated mice with the same disease model. Key Findings Two silencing molecules tested: Both cut alpha-synuclein in the brain …
TL;DR: In a phase III trial of 34 children with a rare inherited energy disorder, the drug dichloroacetate did not improve parent-rated movement problems more than placebo over 4 months. It did lower a harmful blood acid, lactate, and was well tolerated. Key Findings Main goal missed: Movement scores did not differ from placebo (p …
TL;DR: Among Hispanic and Latino adults in the U.S., people with one particular DNA pattern around the APOE gene were more likely to show steep thinking decline over about 7 years. The well-known APOE4 risk version had a weaker link in the same group. Key Findings 3 DNA profiles: Short DNA sequences near APOE sorted …
TL;DR: Young mice carrying the human APOE4 gene, the biggest genetic risk factor for Alzheimer’s, had overexcited memory circuits months before any memory trouble showed up. Removing APOE4 from their neurons returned those cells to normal. Key Findings Early warning spikes: Young APOE4 mice had more abnormal electrical spikes in parts of the hippocampus. Spikes …
TL;DR: A single long-read DNA test covering 564 genes found 17 of 20 known disease-causing changes in 18 people with Parkinson’s or inherited repeat disorders. It missed three large or repetitive changes, so it needs more work before it could replace several separate tests. Key Findings 17 of 20 found (85%): In people whose genetic …
TL;DR: Ambroxol, an old cough medicine, can boost a key brain enzyme in lab studies, but a review of 20 clinical reports found no proven benefit in Gaucher disease or GBA1-linked Parkinson’s. In a randomized trial of people with GBA1-linked Parkinson’s, ambroxol did not improve thinking-test scores over 1 year; scores slightly favored placebo. Key …
TL;DR: A 2026 mouse study in Nature Communications reported that 10 daily bouts of optogenetic dentate-gyrus stimulation produced weeks-long changes in chromatin, nuclear structure, behavior, and spatial coding, while Cyclin B deletion weakened several effects; this ECT-inspired model does not establish how electroconvulsive therapy works in people. Key Findings Ten stimulation sessions produced the durable …
TL;DR: A 2026 mouse study in Communications Biology found that loss of MGAT5B, a brain-enriched enzyme that branches O-mannose sugars on proteins, widened nodes of Ranvier—the intervals between myelin segments that organize axon signaling—and delayed callosal spike timing. Neuron-targeted MGAT5B shifted pooled node widths lower, but the rescue compared only 2 treated with 3 control …
TL;DR: A 2026 mouse study in Gene Therapy used an adeno-associated virus 9 (AAV9) vector to deliver human FMR1 and reduced sound-triggered seizures, excessive digging, and gamma-frequency electroencephalography (EEG) power in selected dose and route groups. The outcomes came from separate experiments, and high-dose intravenous treatment without immunosuppression caused excess deaths. Key Findings Adolescent seizure …
TL;DR: A 2026 Molecular Psychiatry study found that common genetic influences on sustained attention were partly separable from genetic influences on executive function, using task-based cognition data from the AFFECT study and 23andMe participants. Key Findings More than 20,000 participants contributed task-based cognition data: Final GWAS sample sizes ranged from 10,129 people for lapse recovery …
