Precision gene editing: From proof-of-concept to curative therapies.

Authors

Tongtong Cui, Bojin Li, Bingyu Cai, Hui Wang, Wei Li

Published in

Trends in molecular medicine. Jun 30, 2026. Epub Jun 30, 2026.

Abstract

Gene therapy is evolving from gene addition to precise genome editing, enabling the direct correction of disease-causing mutations. Breakthrough technologies, such as clustered regularly interspaced short palindromic repeats-CRISPR-associated protein (CRISPR-Cas) nucleases, base editors, prime editors, and CRISPR-associated transposases are reshaping the therapeutic landscape. This review covers the progression of precision editing technologies and their clinical applications, spanning from ex vivo therapies to in vivo treatments targeting vital organs. The rise of personalized medicine, highlighted by therapies, such as carbamoyl phosphate synthetase 1 editing, underscores the shift toward N-of-1 medicine for rare diseases. Clinical trial progress, delivery and accessibility challenges, and the role of AI in optimizing editing tools and predicting outcomes are also discussed. These innovations are transforming genetic medicine, offering the promise of safer, more durable, and personalized cures.

PMID:
42379944
Bibliographic data and abstract were imported from PubMed on 01 Jul 2026.

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