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Gene Therapy

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

Genome-Wide CRISPR Screen Identifies Two Human Genes That Boost Nonviral Editing

Transient reduction of GJB2 or BET1L activity offers a path to boost lipid-nanoparticle delivery of base editors pending animal and safety studies.

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

NHS Gene Therapy Restores Sight for 6-Year-Old

Oslo Patient Is 10th Documented HIV Remission After CCR5 Stem-Cell Transplant

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

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