Research Articles
Restoring Vision: How Gene Therapy is Revolutionizing Treatment for Retinal Ciliopathies
Explore how gene therapy is transforming treatment for retinal ciliopathies, genetic disorders affecting cellular antennae in the eyes that cause progressive vision loss.
Gene Therapy for Cardiovascular Disease: Rewriting the Code of Heart Health
Explore how gene therapy is revolutionizing cardiovascular medicine through CRISPR technology, clinical trials, and innovative delivery systems.
The Sound of Breakthroughs: Gene Therapy's Promise for Hearing Loss
Explore how gene therapy is revolutionizing treatment for genetic hearing loss, restoring biological hearing for the first time in human history.
Gene Therapy: The Power of Persistence
Explore the latest breakthroughs in gene therapy, including AI-enhanced CRISPR tools and revolutionary treatments for genetic diseases.
Unlocking the Cell: How a Simple Inhibitor Supercharges Gene Therapy
Discover how TBK1 inhibitors dramatically boost gene therapy efficiency by interfering with cellular protein p62, opening new doors for genetic disease treatment.
Gene Therapy: The View from NCATS
Exploring how NCATS is accelerating gene therapy development for rare diseases through innovative platform approaches and collaborative science.
Getting to the Root of It All: Regenerating Hearing and Balance with Gene and Stem Cell Therapies
Explore groundbreaking advances in gene and stem cell therapies for treating hearing and balance disorders, including clinical trials and future directions.
Gene Therapy for Familial Hypercholesterolemia: A Single-Shot Cure on the Horizon?
Explore revolutionary gene therapy approaches for Familial Hypercholesterolemia, including gene silencing and CRISPR-based editing, offering potential long-term solutions for this genetic disorder.
The Gene Therapy Gap: Why Cutting-Edge Science Needs Public Trust to Revolutionize Medicine
Exploring the disconnect between scientific progress in gene therapy and public acceptance, with data-driven insights into perceptions and strategies to bridge the gap.