Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a functional copy. Alternatively DNA encoding a therapeutic protein drug may be introduced.Featured...
A one-time gene-editing therapy restored motor function and prolonged lifespan in mice, spurring further research to refine and optimize the approach for future clinical use. Karen O’Leary News & Views | 16 February 2023 CRISPR gene-editing therapies for hypertrophic cardiomyopathy Pre-symptoma...
You can also make a donation by PayPal and support the Gene Therapy Net website. Most Popular Items Breaking Gene Therapy News Diseases Treated by Gene Therapy Types of Gene Therapy Adeno-Associated Viral Vectors Glossary of Gene Therapy Terms ...
Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research has found. The University of Bristol-led study, published in Molecular Therapy, reveals how age ... Feb 10, 2025 ...
Precision gene therapy for liver disorders increases number of correctly repaired cells Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or inactivating genes. Precisely fixing disease-causing mutations is far more difficult. This ...
Brochure Solutions Products News & events Resources & support Connect every phase of your cell and gene therapy journey We connect you to end-to-end cell and gene therapy solutions that help you deliver life-changing therapies. Our mission is to provide you with connections to help you reach ...
Gene Therapy Latest News Study demonstrates visual acuity improvements following gene therapy for AIPL1-associated inherited retinal dystrophies By Hattie Hayes February 28th 2025 Researchers indicated that pediatric patients with Leber congenital amaurosis experienced improvements in vision. Efficacy and safety...
Brochure Solutions Products News & events Resources & support Connect every phase of your cell and gene therapy journey We connect you to end-to-end cell and gene therapy solutions that help you deliver life-changing therapies. Our mission is to provide you with connections to help you reach...
Data from a phase 2 pharmacodynamic study of ABBV-RGX-314 gene therapy were presented at ASRS 2024. An investigational single-administration gene therapy that delivers a transgene for a soluble anti-vascular endothelial growth factor (VEGF) Fab, the wet AMD therapy is in currently being examined ...
Gene therapy newsNo Abstract available for this article.doi:10.2165/00128413-199610560-00021NoneSpringer International PublishingInpharma Weekly