Over Half of Infants With Genetic Epilepsy May Benefit From ASO Therapy
A multinational study finds 56% of genetically diagnosed infants with epilepsy have variants potentially amenable to antisense oligonucleotide treatment.
20 articles
A multinational study finds 56% of genetically diagnosed infants with epilepsy have variants potentially amenable to antisense oligonucleotide treatment.
Scientists used stem cell-derived vesicles to deliver light-activated gene therapy to the brain without surgery, reversing cognitive decline in Alzheimer's mice.
Researchers combined ultrasound with gene therapy to treat Leigh syndrome, significantly extending survival in mouse models.
New peptide delivery system restores brain connections and memory function in Alzheimer's models, offering hope for treating cognitive decline.
Synthetic peptide chimeras degrade amyloid-beta across the blood-brain barrier, improving cognition in mice with fewer risks than current immunotherapies.
Breakthrough antisense therapy demonstrates sustained benefits for rare genetic form of ALS in extended clinical trial.
Overexpressing Sox9 in brain support cells triggers plaque cleanup and protects cognition in Alzheimer's mouse models.
OSK gene therapy targeting engram neurons restored memory to youthful levels in aged mice and Alzheimer's models, reversing senescence hallmarks.
Microglia package healthy mitochondria into vesicles and deliver them to astrocytes, sharply reducing cognitive decline in tau-pathology mice.
New study identifies KCTD20 gene suppression as a potential therapeutic target for clearing harmful tau proteins in neurodegenerative diseases.
New research reveals combined treatments targeting multiple brain pathways outperform traditional single-drug strategies for Alzheimer's disease.
A boy with PLCB1-related epileptic encephalopathy showed improved seizure control and stabilized brain atrophy after high-dose enteral myo-inositol supplementation.
New research reveals how TSC2 mutations suppress translation of autism and epilepsy genes, offering potential therapeutic targets.
Researchers develop novel immunotherapy using modified brain cells to target Alzheimer's pathology directly in the brain.
Scientists develop pathway-selective drug that activates beneficial brain receptors while avoiding negative feedback loops.
New brain organoid study shows autophagy-boosting compounds can clear harmful tau proteins and rescue synapses in Alzheimer's disease models.
New review reveals promising tau-targeting therapies showing cognitive benefits and slowed brain pathology in Alzheimer's trials.
Researchers modified astrocytes with chimeric antigen receptors to target amyloid plaques, showing promise for Alzheimer's treatment.
After ischemic stroke, a mislocated brain water channel (AQP4) disrupts waste clearance. Blocking AQP4 with TGN-020 corrects its position and restores glymphatic flow.
A newly identified proteasome activator, PA200, clears toxic alpha-synuclein aggregates and maintains function even when standard protein-disposal systems fail.