FeaturedGeneticsNeurologyNeuroscience·June 10, 2019·7 min read‘Like Looking at a Miracle’: Baby Blossoms Thanks to Gene Therapy For Spinal Muscular AtrophyZolgensma, 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
FeaturedGeneticsNeurologyOpen Neuroscience Articles·September 9, 2016·5 min readActivity of Huntington’s Disease Gene Curbed for 6 Months: Mouse StudyResearchers have identified a protein that can turn off the Huntingtin gene's expression.Read More
FeaturedGeneticsNeuroscience·July 3, 2023·6 min readAI Predicts CRISPR’s RNA-Targeting Effects, Revolutionizing Gene TherapyResearchers 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
FeaturedGeneticsNeurologyOpen Neuroscience Articles·October 10, 2016·6 min readAlzheimer’s Could be Treated With Gene Therapy: Mouse StudyResearchers 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 readAn Artificial Retina With the Capacity to Restore Normal VisionResearchers decipher the retina's neural code for brain communication to create novel, more effective prosthetic retinal device for blindness.Read More
FeaturedNeuroscienceOpen Neuroscience Articles·February 9, 2022·5 min readAstrocytes Display Unique Activity PatternsAstrocytes may play a significant role in information processing and memory, a new study reports.Read More
FeaturedGeneticsNeuroscience Videos·April 23, 2014·3 min readBionic Ear Technology Used for Gene TherapyResearchers use electrical pulses delivered from cochlear implants to deliver gene therapy and regrow auditory neurons.Read More
FeaturedNeurology·January 22, 2013·4 min readDiscovering the “Needle in a Haystack” for Muscular Dystrophy PatientsScientists 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
FeaturedGeneticsNeurology·February 19, 2021·4 min readFirst-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer’s DiseaseResearchers 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
Brain CancerFeaturedGeneticsOpen Neuroscience Articles·August 17, 2020·2 min readGene Targeting Helps Overcome the Resistance of Brain Cancer to TherapyMouse study reveals the deletion of the OSMR gene significantly improves glioblastoma tumor response to therapy and extended lifespan.Read More
FeaturedGenetics·December 9, 2015·4 min readGene Therapy Could Extend Estrogen’s Protective Effects on MemoryResearchers use gene therapy to help extend the effectiveness of estrogen for retaining memory.Read More
FeaturedGenetics·November 15, 2015·6 min readGene Therapy For Brain Disorders Delivered by Eye DropsResearchers have developed a simplified approach for delivering and monitoring gene therapy for brain disorders.Read More