Gene editing

Visitors are invited to explore galleries highlighting key discoveries and transformative insights in biomedicine.

RNA-guided enzymes are more diverse and widespread than previously believed

Converting a pathogenic hemoglobin gene to a benign variant enables healthy blood cell production in an animal model of sickle cell disease.

Correcting the mutation that causes progeria with base editing leads to strong symptom reduction and longer lifespan in an animal model

Scientists apply the “Perturb-Seq” method to a living organism for the first time

An engineered bacterial toxin is a key part of a gene editor that can make single-base changes in human mitochondria.

BE-Hive can tell which base editor performs best to repair thousands of disease-causing mutations

By showing they could repair a single recessive mutation behind a form of hearing loss, researchers open the door to using base editing to address a broader range of conditions

A new adenine base editor expands the range of genetic sites that can be edited in human cells, with speed and efficiency far surpassing the original editor.