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Gene Editing

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Life Biosciences Doses First Patient With Eye Reprogramming Gene Therapy

The study uses a doxycycline-controlled three-gene system to treat glaucoma with a reversible safety switch to limit gene activity.

ZNF804A Alteration Boosts Local Protein Production and Synapse Formation in Developing Human Neurons

CRISPR Enzyme Cas12a2 Selectively Destroys Cells Based on RNA in Lab Tests

High-Resolution Map of Pancreatic Cell DNA Methylation Ties ONECUT2 to Type 2 Diabetes

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

CRISPR-Edited Fungus Delivers Faster, Lower-Impact Mycoprotein

FDA Outlines 'Plausible-Mechanism' Pathway to Speed Bespoke Therapies

Single-Infusion CRISPR Therapy Lowers LDL and Triglycerides in First Human Trial

Intellia Pauses Phase 3 Trials of CRISPR Therapy After Severe Liver Event

CNIO Releases 'Human REPAIRome' Mapping 20,000 DNA Repair Scars

St. Jude Screen Rapidly Optimizes CAST Editors With Higher Precision and Activity

Custom CRISPR Base Editor Corrects ACTA2 Mutation in Mice, Extending Survival Fourfold

MD Anderson Debuts PreCiSE, a Genome-Wide CRISPR Platform for Primary NK Cells

Phenol-Modified Lipids Improve mRNA Therapeutics by Cutting Inflammation

CRISPR Epigenome Editing Controls Inflammation and Tumor Growth in Leukemia Cells

AP1M1 Knockout Validated to Enhance Antisense Drug Delivery, Guiding Broader RNA Therapy Development

Fertile Mice With Two Fathers Produce Their Own Offspring

Precision Base Editor Restores Mitochondrial DNA in Patient Cells

University of Geneva Identifies Protein Gatekeepers for Targeted Cancer Therapies

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP