Science ❯ Biology ❯ Genetics

Gene Therapy

Clinical Trials Adeno-Associated Virus Clinical Research Clinical Applications Gene Delivery Systems CRISPR Technology OTOF Gene Sickle Cell Disease Cardiomyocytes Photoreceptor Function

CRISPR Therapeutics’ Pipeline Pitched as Path to One-Time Heart Risk Treatments

Backers see CTX310, CTX320 as single-dose edits that could replace lifelong lipid drugs.

FDA Approves Novartis’ Itvisma for SMA Patients Ages 2 and Up

World-First Gene Therapy for Hunter Syndrome Shows Early Gains in First Child

Prime Editing Tool PERT Debuts as Broad, One-Time Strategy for Nonsense Mutations

Casgevy Launch Stumbles, Yet Analysts Eye Upside for CRISPR Therapeutics

Reactivated Fetal Gene Triggers Division of Adult Human Heart Cells in Lab

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Gene Therapy Reports 75% Slower Huntington’s Progression in 36-Month 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 Three-Year Trial

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

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

First Patient With Usher Syndrome Regains Sight Through Double-Vector Gene Therapy

Global Team Releases Most Complete Human Pangenome and Maps 175,000 Structural Variants

Prime Editing Reverses Rare Childhood Brain Disorder by Correcting Multiple Mutations in Mice

Single Gene Therapy Injection Reverses Deafness Across Age Groups

Experimental Fabry Gene Therapy Achieves Near-Break-Even Costs and Advances to Larger Study

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

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

Penn Researchers Develop Gene Therapy Tools for Advanced Retinal Degenerations