Related Experiment Video
Updated: Jul 2, 2026

CIRCLE-Seq for Interrogation of Off-Target Gene Editing
Published on: November 1, 2024
Precision gene editing: From proof-of-concept to curative therapies
Tongtong Cui1, Bojin Li2, Bingyu Cai2
1Key Laboratory of Organ Regeneration an Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China; Beijing Institute for Stem Cell and Regenerative Medicine, Beijing 100101, China.
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.
Related Concept Videos
CRISPR
Gene Therapy
Gene Therapy
CRISPR/Cas9 Genome Editing
What is Genetic Engineering?
Pharmacogenomics: Identification of New Drug Targets

