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gene therapy

Featured Genetics Neurology Neuroscience

·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.

Image shows a brain slice from a huntington's patient.

Featured Genetics Neurology Open 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.

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.

Image shows brain cells from a mouse cortex.

Featured Genetics Neurology Open 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.

Featured Neuroscience

·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.

Featured Neuroscience Open 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.

This image shows regenerated auditory neurons using gene therapy.

Featured Genetics Neuroscience 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.

Featured Neurology

·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.

This shows the outline of a head and a question mark

Featured Genetics Neurology

·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.