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Gene Therapy

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

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

The agency signaled that Phase I/II results versus external controls would not provide the primary evidence for a BLA.

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