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Neurogenomics – News and Features

Gold, star shapes representing neurons.
News

Newly Identified Neural Stem Cells Could Transform Parkinson's Treatment

The detection of peripheral neural stem cells could transform the treatment of Parkinson's disease and spinal cord injuries.
Close-up of a detailed model of the human brain, used to illustrate neurodevelopmental disorders.
News

New Genetic Cause of Neurodevelopmental Disorders Identified

Researchers at the Icahn School of Medicine have discovered mutations in the non-coding gene RNU2-2 as a new cause of neurodevelopmental disorders. This highlights the role of non-coding genes in these conditions.
A mother playing with her child with Down syndrome.
News

First Down Syndrome Participant Dosed in Trial of Alzheimer’s Treatment

Researchers have dosed the first participant in a clinical trial of an investigational medicine designed to lower the amount of amyloid precursor protein for the potential treatment of Alzheimer’s disease.
Digitally rendered neurons with glowing synapses, illustrating brain activity related to Tourette’s syndrome.
News

Brain Study Reveals Key Cellular Changes Behind Tourette Syndrome

A new study provides insights into Tourette syndrome by analyzing individual brain cells. Researchers found key changes, including a loss of interneurons, metabolic stress in medium spiny neurons, and increased inflammatory activity in microglia.
A woman experiencing abdominal pain while lying on a couch, highlighting the need for pain relief.
News

Estrogen and Progesterone Regulate Natural Opioids in Women

Researchers at UCSF discovered that regulatory T cells (T-regs) in the meninges produce natural opioids, and their pain-suppressing effects are influenced by estrogen and progesterone.
A flowerpot snake slithering across the ground, surrounded by autumn leaves.
News

How the Flowerpot Snake’s Unique DNA Repair Mechanism Works

Researchers at the University of Texas at Arlington discovered how the flowerpot snake, with three sets of chromosomes, can reproduce without a mate. The study uncovers the snake’s DNA repair mechanisms.
A group of neurons with electric signals on the synapses.
News

Gene Therapy Restores Motor Capacity in Animal Models of Rare Neurological Disease

A recent study demonstrated the potential of gene therapy to restore motor capacity in an ultra-rare disease, megalencephalic leukoencephalopathy with subcortical cysts (MLC), even when treatment begins after symptom onset.
A single strand of mRNA used in RNA therapeutics such as COVID-19 mRNA vaccines.
Article

The Future of RNA Therapeutics, From Design to Delivery

Discover how RNA therapeutics are reshaping medicine, offering new treatments for previously undruggable targets.
3D rendering of neural and vascular networks, illustrating the complex nervous system affected by ALS (amyotrophic lateral sclerosis).
News

New Model Mimics TDP-43 Aggregation in ALS and Frontotemporal Dementia

Researchers have developed a laboratory model that mimics the pathological aggregation of TDP-43, a key protein in ALS and frontotemporal dementia. The model, based on human cells, recapitulates TDP-43’s mislocalisation and aggregation.
The outside of a US FDA building.
News

FDA Approves First Therapy To Treat Hyperphagia in Prader-Willi Syndrome

Soleno Therapeutics today announced that the US FDA has approved VYKAT XR (diazoxide choline) extended-release tablets, for the treatment of hyperphagia in adults and children four years of age and older with Prader-Willi syndrome.
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