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Gene Therapy

Clinical Trials Personalized Medicine Gene Editing Safety Measures Crispr Technology Neurodegenerative Diseases Breakthroughs OSK System Muscular Dystrophy Mucopolysaccharidosis mRNA Technology HIV Treatment Hearing Restoration Hematopoietic Stem Cell Therapy Gene Expression Drug Delivery Cholesterol Management Rare Genetic Disorders Hemophilia B Huntington's Disease Inherited Deafness mRNA Vaccines Auditory Neuropathy Injectable Gene Therapy Hemophilia B Treatment Adeno-Associated Viruses Deafness Nanocarriers

First Patient Dosed in Human Trial for Cell 'Rejuvenation' Therapy

The FDA-cleared study delivers a controllable gene therapy to the eye to try to restore aged nerve cells while prioritizing safety monitoring.

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

FDA Setback Puts uniQure’s Huntington’s Gene Therapy Filing in Doubt

MD Anderson Debuts PreCiSE, a Genome-Wide CRISPR Platform for Primary NK Cells

HIV Gene Therapy Harnesses Viral RNA to Lock Virus Into Dormant State

Portable Device Allows Hospitals to Make Personalized Gene Therapies On Demand