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Gene Therapy

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

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

The one-time autologous treatment won conditional clearance on biomarker gains, requiring long‑term confirmation of benefit.

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