Unlocking the Potential of Gene Therapy for Genetic Skin Disorders
Original story from the University of British Columbia (British Columbia, Canada). Gene-editing tools like CRISPR have unlocked new treatments for...
genetic
UMass Chan licenses gene therapy for rare genetic disorder to Raiden Science Foundation
Raiden Pham, who was diagnosed with UBA5 disorder in 2021, with his mother Linda Pham. Photo courtesy of Tommy and Linda...
Single protein is key to treating a deadly genetic heart disease targeting young athletes
A research team at the University of California San Diego has discovered a novel and promising method of treating arrhythmogenic...
Gene editing vs gene therapy: Understanding two paths to genetic medicine
How two complementary approaches are reshaping treatment possibilities for inherited and acquired diseases—each with distinct strengths, challenges, and clinical considerations.Gene-based...
New hope for children with devastating rare genetic disorder, thanks to world-first research in Manchester
The parents of a three-year-old boy born with a devastating, life-limiting genetic condition say they are now excited for his...
Experimental gene therapy restores immune system function in children with genetic immune disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained...
New Gene-Editing Tech Holds Promise for Treating Complex Genetic Diseases
Some genetic disorders—such as cystic fibrosis, hemophilia and Tay Sachs disease—involve many mutations in a person’s genome, often with enough...
REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss (Revised)
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study. This gene therapy...
REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.This gene therapy is...
