Gene therapy shows long-term benefits for patients with rare pediatric brain disease
Credit: Pixabay/CC0 Public domain Cerebral adrenoleukodystrophy (CALD) is a rare and progressive genetic brain disease that occurs primarily in young ...
Tag: gene
Novel gene therapy shows promise for targeting epilepsy-linked mutation
Clearance of mutant protein and restoration of normal neuronal activity by Gapmer ASO. Credit: NUS Medicine Researchers at the Yong ...
Discovery of key gene offers new hope for treatment of chronic myelogenous leukemia
Schematic image, showing R-loop resolution by REXO5 (left) and inhibition of R-loop resolution by REXO5 mutation (right). Credit: Leukemia (2024). ...
Comprehensive gene activity mapping brings hope to chronic pain patients
Temporal molecular dynamics of nociceptors and pain states during neuropathic pain. Credit: Natural communications (2024). DOI: 10.1038/s41467-024-52052-8 Researchers at Karolinska ...
Study reveals key role of TRIO gene in epileptic encephalopathy
Defective migration of MGE-derived cortical INs into Trio–/– embryos. Credit: Molecular Psychiatry (2024). DOI: 10.1038/s41380-024-02742-y What are the molecular and ...
Scientists decode a key mutation in many cancers, highlighting the increased role of RNA in human gene expression
Credit: Unsplash/CC0 Public domain Inside every cell, inside every nucleus, your existence depends on an incredibly complicated dance. Proteins are ...
The structure of a eukaryotic CRISPR-Cas homolog, Fanzor2, shows promise for gene editing
Elizabeth Kellogg, PhD, Department of Structural Biology at St. Jude, used cryo-EM to study the evolutionary journey of Fanzor2, a ...
How a protein keeps gene clusters silent in the cell nucleolus
Credit: Scientific advances (2024). DOI: 10.1126/sciadv.ado1662 In a discovery that sheds light on the complex mechanisms of gene regulation, EPFL ...
Single-dose gene therapy could change lives of adults with hemophilia B
Credit: Pixabay/CC0 Public Domain Adults with hemophilia B saw their number of bleeding episodes reduced by an average of 71% ...
Eyesight improves 100-fold after gene therapy trial
Credit: Unsplash/CC0 Public Domain The vision of people with a rare inherited disease that causes vision loss in childhood improved ...
