compact RT editor

The compact RT editor is a miniature genome editing system developed by Arbor Biotechnologies for precise DNA modification. It consists of a miniature nuclease containing RuvC and HNH domains (RHNs) engineered into a nickase and fused to a small, highly active reverse transcriptase (RT). With a total size of under 1200 amino acids, the entire editor and its guide RNA can be packaged into a single adeno-associated virus (AAV) vector, overcoming the packaging limits of first-generation prime editors. The system utilizes template-primed reverse transcription to introduce specific edits without requiring double-strand DNA breaks. It has demonstrated in vivo efficacy in mouse models, achieving significant precise editing in the liver and brain, and is being developed for neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS), Huntington’s Disease, and Alzheimer’s Disease.

Other names

all-in-one AAV compact RT editor