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gene therapy

Auditory Neuroscience Featured Genetics Neuroscience

·July 3, 2025·5 min read

Gene Therapy Restores Hearing in Those with Deafness

A new study shows that gene therapy can significantly improve hearing in both children and adults with congenital deafness caused by mutations in the OTOF gene. The therapy uses a synthetic virus to deliver a healthy copy of the gene directly to the inner ear, with hearing improvements observed in all ten participants within one month.

Autism Featured Genetics Neuroscience

·November 20, 2024·6 min read

Gene Repair Restores Brain Signal Efficiency in Autism

Autism-linked SHANK3 gene mutations disrupt not only neurons but also oligodendrocytes, essential for producing myelin, which insulates nerve fibers. This damage reduces brain signal efficiency and impairs behavior. Using gene therapy, researchers successfully repaired these cells in a mouse model, restoring their function and myelin production. They validated their findings with human-derived stem cells, confirming similar impairments and repair mechanisms.

Featured Neuroscience

·October 10, 2024·5 min read

Controlling Brain Activity with Magnetic Fields

Researchers have developed a breakthrough technology using magnetic fields to control specific brain circuits non-invasively, potentially transforming treatments for conditions like Parkinson’s and depression. This technique, termed "magnetogenetics," delivers gene therapy to target neurons and uses magnetic fields to activate or inhibit them, allowing precise manipulation without invasive implants. In mouse studies, this approach successfully reduced movement issues associated with Parkinson’s. Future research aims to explore clinical applications for psychiatric disorders and chronic pain, providing a new frontier in brain circuit modulation.

Featured Genetics Neuroscience Visual Neuroscience

·September 6, 2024·4 min read

Gene Therapy Restores Vision

A new gene therapy has restored vision in patients with Leber congenital amaurosis type I (LCA1), a rare genetic condition causing blindness. In a small trial, those receiving the highest dose saw up to a 10,000-fold improvement in light sensitivity and significant gains in reading and navigation abilities.

Featured Genetics Neurology Neuroscience

·May 16, 2024·7 min read

Engineered Virus Crosses Blood-Brain Barrier to Deliver Gene Therapy

Researchers have engineered a gene therapy delivery vehicle that efficiently crosses the blood-brain barrier in mice. This breakthrough uses a human protein to ferry therapeutic genes into the brain, potentially paving the way for safer and more effective treatments for various brain diseases, including neurodevelopmental and neurodegenerative disorders.

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.

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 Genetics Neuroscience

·July 3, 2023·6 min read

AI Predicts CRISPR’s RNA-Targeting Effects, Revolutionizing Gene Therapy

Researchers developed a deep learning model, TIGER, that accurately predicts on- and off-target activity of RNA-targeting CRISPR tools. This new approach allows the fine-tuning of gene activity in human cells.

Featured Genetics Neuroscience Visual Neuroscience

·April 26, 2023·3 min read

Gene Therapy for Dogs With Inherited Blinding Eye Disease Set for Human Trials

A groundbreaking gene therapy proven successful in treating an inherited, blinding eye disease in dogs is now poised for clinical trials in humans with the rare condition retinitis pigmentosa. The therapy, which halts vision loss by introducing a normal copy of the CNGB1 gene, could potentially benefit around 2 million people affected worldwide.

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 Neuroscience

·October 21, 2022·5 min read

Using Light Stimulation to Modulate Brain Waves

Researchers have developed a new system that comprises of a brain implant containing LEDs and gene therapy to modulate brain waves. The technology showed success in suppressing abnormal brain waves akin to those seen during an epileptic seizure.