Science ❯ Genetics

Gene Therapy

Clinical Trials CRISPR Technology Otoferlin Gene Huntington's Disease Applications in Medicine Stem Cell Research LAMP2 Gene Follistatin Deafness Personalized Medicine Gene Silencing Neurodegenerative Diseases CCR5 Mutation Disease Mechanisms Skin Conditions Adeno-Associated Virus AADC Deficiency Hearing Impairment FOXP3 Gene CAR-T Therapy Gene Editing Techniques CRISPR Cochlear Implants AAV Technology Regulatory Pathways mRNA Technology Clinical Research Protein Engineering SCN1A Gene Hearing Loss Neuronal Plasticity Sickle Cell Disease

Early Trials of Gene Regulation Drug Cut Dravet Seizures by Up to 91%, NEJM Reports

The antisense therapy boosts SCN1A protein to target the disorder’s genetic cause.

Lilly Strikes Up to $1.12 Billion Pact With Seamless Therapeutics for Hearing‑Loss Gene Editing

Aurora Therapeutics Launches to Scale Bespoke Gene Editing for PKU

FDA Meeting Minutes Undercut uniQure’s Plan to File Huntington’s Gene Therapy for Approval

Regeneron, Tessera Strike $150 Million Gene-Editing Pact for Alpha‑1 Antitrypsin Deficiency

Lowering a Brain Protein Reverses Age-Related Memory Deficits in Mice

FDA Approves Zevaskyn Gene Therapy for Healing Large Epidermolysis Bullosa Wounds

FDA Halts Rocket’s RP-A501 Gene Therapy for Danon Disease After Patient’s Death