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gene therapy

Featured Genetics Neurology Neuroscience

·May 7, 2026·7 min read

Gene Therapy Restores Walking After Paralysis

Researchers have used a designer cytokine, hIL-6, to "rewire" the spinal cord by stimulating the sprouting of new connections from intact nerve fibers. This approach successfully restored coordinated walking in paralyzed mice, highlighting a powerful new strategy for treating spinal cord contusions.

Autism Featured Genetics Neuroscience Open Neuroscience Articles

·May 2, 2022·5 min read

Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids

A brain organoid study reveals how a genetic mutation associated with Pitt-Hopkins syndrome, a profound form of autism, disrupts neural development. Using gene-editing technology, researchers recovered the function of the TCF4 gene and effectively restored neural structure and function.

Autism Featured Genetics Neuroscience

·July 15, 2026·11 min read

Gene Therapy Reverses Severe Traits of Fragile X Syndrome

The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of elevated low-gamma EEG power.

Featured Genetics Neurology Neuroscience

·May 9, 2022·4 min read

Gene Therapy Shows Promise in Treating Neuropathy From Spinal Cord Injuries

Gene therapy that inhibits targeted nerve cell signals effectively improves symptoms of neuropathic pain without negative side effects in mouse models of spinal cord injury or peripheral nerve damage.

Featured Genetics Neurology Neuroscience

·April 27, 2024·7 min read

Gene Therapy Slows ALS Progression

Researchers made a significant breakthrough in ALS treatment using a new gene therapy, marking a notable slow in disease progression for a patient with an aggressive form of ALS. The patient, treated since early 2020, has maintained much of their physical and social abilities, exceeding typical life expectancy and functionality projections for their condition.

This shows a diagram of a normal brain slice and one affected by Alzheimer's disease.

Featured Genetics Neurology Open Neuroscience Articles

·August 27, 2015·4 min read

Gene Therapy Triggers Functional Cell Growth of Degenerating Neurons in Alzheimer’s Brains

According to a new study, degenerating neurons in the brains of Alzheimer's patients measurably responded to a new, experimental gene therapy.

Featured Genetics Neurology Open Neuroscience Articles

·March 19, 2021·3 min read

Gene Therapy Using ‘Zinc Fingers’ May Help Treat Alzheimer’s Disease

A new genetic engineering strategy significantly reduces levels of tau in animal models of Alzheimer's disease. The treatment, which involves a single injection, appears to have long-last effects.

Featured Genetics Neurology Neuroscience

·November 22, 2022·4 min read

Gene-Delivering Viruses Reach the Brain in Step Toward Gene Therapy for Neurological Diseases

Researchers have developed a family of adeno-associated viral vectors (AAVs) that are able to cross the blood-brain barrier to deliver gene therapies directly to the brain.

Featured Genetics Neurology

·July 29, 2020·6 min read

Getting Gene Therapy to the Brain

Researchers successfully applied a gene therapy platform to completely correct brain defects in a large animal model of a human genetic disease.

Auditory Neuroscience Featured Genetics Neuroscience

·August 11, 2023·4 min read

Hearing Loss Reversed Using Gene Therapy

Researchers made a breakthrough in reversing hearing loss in mice. By targeting the inactive Spns2 gene and activating it using a specific enzyme, the team successfully restored the mice's hearing in low and middle frequency ranges.

Featured Neurology Neuroscience

·June 19, 2017·6 min read

How to Stop the Nasty Lurking Toxoplasmosis Parasite? Target Its ‘Stomach’

Study suggests new avenues for treating toxoplasmosis parasite infections.

Diagram of a healthy neuron and one from a Parkinson's patient.

Featured Genetics Open Neuroscience Articles

·October 9, 2015·3 min read

Immune Gene May Prevent Alzheimer’s and Parkinson’s Disease

According to a new study, treatment with interferon-β gene therapy successfully prevents neuron death and disease effects in experimental models of neurodegenerative diseases.

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gene therapy

Gene Therapy Restores Walking After Paralysis

Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids

Gene Therapy Reverses Severe Traits of Fragile X Syndrome

Gene Therapy Shows Promise in Treating Neuropathy From Spinal Cord Injuries

Gene Therapy Slows ALS Progression

Gene Therapy Triggers Functional Cell Growth of Degenerating Neurons in Alzheimer’s Brains

Gene Therapy Using ‘Zinc Fingers’ May Help Treat Alzheimer’s Disease

Gene-Delivering Viruses Reach the Brain in Step Toward Gene Therapy for Neurological Diseases

Getting Gene Therapy to the Brain

Hearing Loss Reversed Using Gene Therapy

How to Stop the Nasty Lurking Toxoplasmosis Parasite? Target Its ‘Stomach’

Immune Gene May Prevent Alzheimer’s and Parkinson’s Disease

PCOS/PMOS Increases Alzheimer’s Risk

Alcohol Tricks Brain Cells Into Destroying Themselves

Daytime Napping Linked to Higher Risk of Dementia

Prenatal Stress Alters the Placenta to Shape Fetal Brain Development

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