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Gene Therapy

Clinical Trials Adeno-Associated Virus Gene Delivery Systems Clinical Applications Sickle Cell Disease OTOF Gene Clinical Research CRISPR Technology Advances in Gene Therapy KCNJ2 as a Protective Factor Stem Cell Research Klotho Protein CPS1 Deficiency AAV Vectors Neurodegenerative Diseases RNA Technology Deafness Duchenne Muscular Dystrophy OTOF Gene Therapy Spinal Muscular Atrophy Ophthalmology Usher Syndrome Therapeutic Applications Haploinsufficiency Huntington's Disease Craniosynostosis CAR-T Therapy Kallikrein Inhibition SYNGAP1-related Disorders Adenosine Deaminase Deficiency Cyclin A2 CRISPR-Cas9 Personalized Medicine Genome Editing Enzyme Deficiency SMN1 Gene Stem Cell Treatment Lentiviral Vectors SCN1A Gene Rare Diseases Viral Vectors Adeno-Associated Viruses Cardiomyocytes Cardiac Function Photoreceptor Function CCR5 Mutation

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

Regulators based the decision on 52-week motor gains with a consistent safety profile.

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

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

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

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

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