Design of highly functional genome editors by modelling CRISPR-Cas sequences.

ABSTRACT: Gene editing has the potential to solve fundamental challenges in agriculture, biotechnology and human health. CRISPR-based gene editors derived from microorganisms, although powerful, often show notable functional tradeoffs when ported into non-native environments, such as human cells¹. Artificial-intelligence-enabled design provides a powerful alternative with the potential to bypass evolutionary constraints and generate editors with optimal properties. Here, using large language models² trained on biological diversity at scale, we demonstrate successful precision editing of the human genome with a programmable gene editor designed with artificial intelligence. To achieve this goal, we curated a dataset of more than 1 million CRISPR operons through systematic mining

SUBMITTER: Ruffolo JA 

PROVIDER: S-EPMC12422970 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature