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Gene Therapy

Clinical Trials Genetic Disorders Huntington's Disease Phage Therapy Personalized Medicine Gene Editing Cardiovascular Health CRISPR Technology Hearing Impairment Duchenne Muscular Dystrophy

Engineered Mini CRISPR Achieves Up to 90% Editing in Human Cells

The compact enzyme fits into adeno-associated virus vectors used to target gene editors to specific tissues.

REGENXBIO Investors Face April 14 Deadline to Seek Lead Role in RGX-111 Securities Suit

Sarepta Reports 3-Year EMBARK Data Showing Elevidys Slows Duchenne Progression

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

India Unveils First Homegrown Antibiotic Nafithromycin

Enzyme Method Turns Donor Kidney Into Universal Type O in First Human Test

Gene Therapy Shows 75% Slower Huntington’s Decline in Early Trial

uniQure Gene Therapy Shows 75% Slowing of Huntington’s in Early Trial

uniQure’s AMT-130 Slows Huntington’s Progression by 75% at 36 Months in Early Trial

Gene Therapy Slows Huntington’s Progression by 75% in Early Trial, uniQure Says

AI Designs Functional Bacteriophage Genomes That Kill E. Coli

Researchers Debut Three Precision Gene Editing Tools Reducing Damage and Off-Target Errors

David Baltimore, Nobel Laureate Who Discovered Reverse Transcriptase, Dies at 87

DNA-Coated Nanoparticles Triple CRISPR Editing in Lab Tests

Small Subset of Newborn Liver Cells Drives Growth, Recasting Pediatric Gene Therapy Timing

Yale’s ChronODE Pinpoints Gene Activation Windows in the Developing Brain

Kidney Organoids Reveal NFκB‑Linked AAV2 Toxicity, With Bardoxolone Methyl Mitigating Damage

AI-Guided Pythia Steers CRISPR Edits Toward Precise DNA Integration

FDA Reinstates Biologics Director Vinay Prasad Less Than Two Weeks After Resignation

Single Birth Injection Protects Primates From HIV, Paves Way for Newborn Safety Trials