Health ❯ Medical Research

Gene Therapy

Clinical Trials Genetic Disorders Huntington's Disease Cardiovascular Health CRISPR Technology Personalized Medicine Phage Therapy Ophthalmology Duchenne Muscular Dystrophy Gene Editing Hearing Impairment Gene Manipulation MicroRNA Treatments Targeted Therapy Adeno-Associated Virus (AAV) mRNA Vaccines AFM-Téléthon Sickle Cell Disease Heart Failure Epigenetic Editing OTOF Gene OTOF Gene Therapy AAV Vectors Neurological Diseases Acoustic Stimulation Liver Diseases Antisense Oligonucleotides Neuroscience RNA Interference (RNAi) Hearing Loss DNA Repair Congenital Deafness Hearing Disorders X-SCID Treatment HIV Gene Therapy Cancer Research Cytokine Research Safety Testing Cell Therapy Therapeutic Applications Pediatric Gene Therapy Neurodegenerative Diseases Neurological Disorders Genetic Modifications Genome Editing Techniques Infectious Diseases Gene Silencing MicroRNA Tumor Angiogenesis Hearing Restoration Diabetic Kidney Disease Cancer Treatment Cardiomyopathy Inherited Disorders Biotechnology Non-Viral Techniques Funding Challenges Clinical Evaluation Quality Control CRISPR Applications Blood Disorders RNA Interference Inherited Conditions Viral Vectors siRNA Therapy Sarepta Therapeutics MicroRNA Applications mRNA Technology

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

The readout lands under heightened FDA scrutiny following two liver‑failure deaths in non‑ambulatory patients.

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

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

uniQure Gene Therapy Shows 75% Slowing of Huntington’s 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

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

Active Interstellar Comet 3I/ATLAS Set for Safe Solar System Passage

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

Eli Lilly to Acquire Verve Therapeutics for $1.3 Billion

Study uncovers early-life window for in vivo gene therapy of blood stem cells

NIH Unveils AI-Driven Gene Delivery Toolkit for Precise Neural Targeting