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          gene therapy

          This shows baby Arabella Smygov following gene therapy treatment for SMA
          FeaturedGeneticsNeurologyNeuroscience
          ·June 10, 2019·7 min read

          ‘Like Looking at a Miracle’: Baby Blossoms Thanks to Gene Therapy For Spinal Muscular Atrophy

          Zolgensma, an FDA approved gene therapy, supplements the production of the SMN protein to improve motor neuron function in children with spinal muscular atrophy after just one dose.
          Read More
          Image shows a brain slice from a huntington's patient.
          FeaturedGeneticsNeurologyOpen Neuroscience Articles
          ·September 9, 2016·5 min read

          Activity of Huntington’s Disease Gene Curbed for 6 Months: Mouse Study

          Researchers have identified a protein that can turn off the Huntingtin gene's expression.
          Read More
          This shows RNA.
          FeaturedGeneticsNeuroscience
          ·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.
          Read More
          Image shows brain cells from a mouse cortex.
          FeaturedGeneticsNeurologyOpen Neuroscience Articles
          ·October 10, 2016·6 min read

          Alzheimer’s Could be Treated With Gene Therapy: Mouse Study

          Researchers report they have successfully prevented the development of Alzheimer's in mice by using gene therapy delivered by a virus to the brain.
          Read More
          FeaturedNeuroscience
          ·August 14, 2012·5 min read

          An Artificial Retina With the Capacity to Restore Normal Vision

          Researchers decipher the retina's neural code for brain communication to create novel, more effective prosthetic retinal device for blindness.
          Read More
          This shows a diagram of an astrocyte
          FeaturedNeuroscienceOpen Neuroscience Articles
          ·February 9, 2022·5 min read

          Astrocytes Display Unique Activity Patterns

          Astrocytes may play a significant role in information processing and memory, a new study reports.
          Read More
          This image shows regenerated auditory neurons using gene therapy.
          FeaturedGeneticsNeuroscience Videos
          ·April 23, 2014·3 min read

          Bionic Ear Technology Used for Gene Therapy

          Researchers use electrical pulses delivered from cochlear implants to deliver gene therapy and regrow auditory neurons.
          Read More
          This shows neurons.
          FeaturedNeuroscience
          ·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.
          Read More
          Dongsheng Duan is shown in the image.
          FeaturedNeurology
          ·January 22, 2013·4 min read

          Discovering the “Needle in a Haystack” for Muscular Dystrophy Patients

          Scientists identified a sequence in the dystrophin gene that is essential for helping muscle tissues function, a breakthrough discovery that could lead to treatments for the deadly hereditary disease.
          Read More
          This shows a virus and neurons.
          FeaturedGeneticsNeurologyNeuroscience
          ·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.
          Read More
          This shows the outline of a head and a question mark
          FeaturedGeneticsNeurology
          ·February 19, 2021·4 min read

          First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer’s Disease

          Researchers launch the first-in-human phase 1 clinical trial to assess the safety and efficacy of a gene therapy which will deliver BDNF to the brains of those with Alzheimer's and mild cognitive impairment.
          Read More
          This shows a brain.
          AutismFeaturedGeneticsNeuroscience
          ·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.
          Read More
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          A 3D digital rendering showing a developing fetal brain connected by glowing neural pathways and floating DNA strands, surrounded by subtle immune cells.

          How Severe Illness During Pregnancy Alters Fetal Brain Development

          A 3D digital illustration of a human brain side-by-side comparison, highlighting the language centers glowing brightly and equally in both a younger and older brain, while executive function areas are dimmed in the older brain.

          Aging Brains Maintain Robust Language Networks Despite Cognitive Decline

          Rejuvenation of Microglia by Youth Protein TIMP2 in the Aging Brain

          Youth Protein TIMP2 Restores Immune Function in the Aging Brain

          A multi-panel documentary photo. Left: A Black man sits alone on a locker room bench with his head down. Center: A Latino man sits withdrawn at a family kitchen table. Right: A White man looks thoughtfully at wellness pamphlets in a public waiting area. All convey isolation.

          Shame, Stigma, and Culture Prevent Male Domestic Violence Survivors From Seeking Help

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