Prime editing for precise and highly versatile genome manipulation

PJ Chen, DR Liu - Nature Reviews Genetics, 2023 - nature.com
Programmable gene-editing tools have transformed the life sciences and have shown
potential for the treatment of genetic disease. Among the CRISPR–Cas technologies that …

CRISPR/Cas9 therapeutics: progress and prospects

T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - … and Targeted Therapy, 2023 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

Genotoxic effects of base and prime editing in human hematopoietic stem cells

M Fiumara, S Ferrari, A Omer-Javed, S Beretta… - Nature …, 2024 - nature.com
Base and prime editors (BEs and PEs) may provide more precise genetic engineering than
nuclease-based approaches because they bypass the dependence on DNA double-strand …

CRISPR in cancer biology and therapy

A Katti, BJ Diaz, CM Caragine, NE Sanjana… - Nature Reviews …, 2022 - nature.com
Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

Therapeutic in vivo delivery of gene editing agents

A Raguram, S Banskota, DR Liu - Cell, 2022 - cell.com
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing

AV Anzalone, XD Gao, CJ Podracky, AT Nelson… - Nature …, 2022 - nature.com
The targeted deletion, replacement, integration or inversion of genomic sequences could be
used to study or treat human genetic diseases, but existing methods typically require double …

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

S Banskota, A Raguram, S Suh, SW Du, JR Davis… - Cell, 2022 - cell.com
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …

Engineered pegRNAs improve prime editing efficiency

JW Nelson, PB Randolph, SP Shen, KA Everette… - Nature …, 2022 - nature.com
Prime editing enables the installation of virtually any combination of point mutations, small
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …

Efficient prime editing in mouse brain, liver and heart with dual AAVs

JR Davis, S Banskota, JM Levy, GA Newby… - Nature …, 2024 - nature.com
Realizing the promise of prime editing for the study and treatment of genetic disorders
requires efficient methods for delivering prime editors (PEs) in vivo. Here we describe the …

Precise integration of large DNA sequences in plant genomes using PrimeRoot editors

C Sun, Y Lei, B Li, Q Gao, Y Li, W Cao, C Yang… - Nature …, 2024 - nature.com
A technique for chromosomal insertion of large DNA segments is much needed in plant
breeding and synthetic biology to facilitate the introduction of desired agronomic traits and …