gene therapy News
Latest articles and news about gene therapy on AXL Media.
Latest Articles
- Whitehead Institute Researchers Re-engineer Human Producer Cells to Enhance Potency of Gene Editing Delivery Vehicles
Published: Apr 24, 2026
Section: Medical News
A new study published in Nature Communications reveals a platform for optimizing the cellular factories used to create virus-like particles for gene editing. By identifying and dis...
- International Trial Restores Hearing in Patients With Genetic Deafness Through Breakthrough OTOF Gene Therapy
Published: Apr 23, 2026
Section: Health
A landmark multicenter study co-led by Mass Eye and Ear and Fudan University has successfully restored hearing in approximately 90% of participants suffering from DFNB9, a rare for...
- Wake Forest Scientists Identify Shared Genetic Blueprint for Limb Regeneration Across Axolotls, Fish, and Mice
Published: Apr 17, 2026
Section: Research
A multi-institutional study published in the Proceedings of the National Academy of Sciences has identified "SP genes" as universal drivers of limb regeneration in diverse species....
- UCD researchers identify cellular courier system capable of bypassing natural biological barriers for drug delivery
Published: Apr 16, 2026
Section: Research
Scientists at University College Dublin have discovered a "condensate corona" mechanism that allows cells to exchange complex biological instructions via specialized droplets. This...
- Clinical Transition of CRISPR Gene Therapies Requires Multidisciplinary Non-Clinical Safety Framework
Published: Apr 11, 2026
Section: Health Technology
A new review published in Genes & Diseases outlines the rigorous non-clinical safety assessments essential for advancing CRISPR/Cas9 technologies from the lab to human patients. Ex...
- Virica Biotech and FUJIFILM Biosciences Partner to Scalably Boost Viral Vector Yields for Gene Therapies
Published: Apr 9, 2026
Section: Medical News
Virica Biotech has entered a strategic collaboration with FUJIFILM Biosciences to optimize viral sensitizer formulations for Adeno-Associated Virus (AAV) production using the Balan...
- USC Physician-Scientist Mohamed Abou-el-Enein Secures Rare Double Honors From American Society of Gene + Cell Therapy
Published: Apr 1, 2026
Section: Science & Tech
Dr. Mohamed Abou-el-Enein has been named the 2026 Outstanding New Investigator by the ASGCT for his decade of contributions to translational medicine. His research team also receiv...
- University of Michigan Engineers Develop Protein Nanoparticles to Deliver Gene Therapy Without the Risks of Viral Vectors
Published: Mar 31, 2026
Section: Research
Researchers at the University of Michigan have successfully used protein-based nanoparticles to genetically modify human liver, kidney, and immune cells. This "artificial virus" al...
- Mount Sinai Researchers Identify Recessive ReNU2 Syndrome: The Most Prevalent Recessive Neurodevelopmental Disorder to Date
Published: Mar 30, 2026
Section: Research
Scientists at the Icahn School of Medicine at Mount Sinai have discovered a previously hidden genetic condition caused by mutations in the non-coding gene RNU2-2. Estimated to acco...
- FDA Approves Landmark Gene Therapy for Severe LAD-I After Successful UCLA-Led Clinical Trials
Published: Mar 28, 2026
Section: Health
The U.S. Food and Drug Administration has approved Kresladi, the first-ever gene therapy for leukocyte adhesion deficiency-I (LAD-I), a rare and often fatal pediatric immune disord...
- FDA Grants Landmark Approval to Kresladi as First Gene Therapy for Rare Pediatric Immune Disorder LAD-I
Published: Mar 28, 2026
Section: Health
The U.S. Food and Drug Administration has approved Kresladi, a groundbreaking one-time gene therapy developed by Rocket Pharmaceuticals and UCLA researchers for Leukocyte Adhesion...
- University of Chicago and IDefine Launch Rapid Response Research Program to Engineer First-of-its-Kind Molecular Therapy for Kleefstra Syndrome
Published: Mar 27, 2026
Section: Science & Tech
The University of Chicago Department of Chemistry has partnered with the advocacy group IDefine to launch a six-month intensive research program targeting Kleefstra syndrome (KLEFS...