Health ❯ Medical Research

Gene Therapy

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

AlphaFold3-Guided ContactSeek Rewrites Base Editors to Cut Off-Target Edits

It offers a fast route to safer base editors by using AlphaFold3 to map and redesign protein–DNA contacts.

Cambridge Team Uses Base Editing to Knock Out NANOG and Expose Limits of Embryo Editing

CRISPR Enzyme Cas12a2 Selectively Destroys Cells Based on RNA in Lab Tests

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

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