Therapeutic in vivo delivery of gene editing agents
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 …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases
MTN Yarnall, EI Ioannidi, C Schmitt-Ulms… - Nature …, 2023 - nature.com
Programmable genome integration of large, diverse DNA cargo without DNA repair of
exposed DNA double-strand breaks remains an unsolved challenge in genome editing. We …
exposed DNA double-strand breaks remains an unsolved challenge in genome editing. We …
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …
technologies has opened up the possibility of directly targeting and modifying genomic …
Walking a tightrope: the complex balancing act of R-loops in genome stability
JR Brickner, JL Garzon, KA Cimprich - Molecular cell, 2022 - cell.com
Although transcription is an essential cellular process, it is paradoxically also a well-
recognized cause of genomic instability. R-loops, non-B DNA structures formed when …
recognized cause of genomic instability. R-loops, non-B DNA structures formed when …
Untangling the roles of TOP2A and TOP2B in transcription and cancer
Type II topoisomerases (TOP2) are conserved regulators of chromatin topology that catalyze
reversible DNA double-strand breaks (DSBs) and are essential for maintaining genomic …
reversible DNA double-strand breaks (DSBs) and are essential for maintaining genomic …
The molecular basis and disease relevance of non-homologous DNA end joining
B Zhao, E Rothenberg, DA Ramsden… - … Reviews Molecular Cell …, 2020 - nature.com
Non-homologous DNA end joining (NHEJ) is the predominant repair mechanism of any type
of DNA double-strand break (DSB) during most of the cell cycle and is essential for the …
of DNA double-strand break (DSB) during most of the cell cycle and is essential for the …
Intertwining roles of R-loops and G-quadruplexes in DNA repair, transcription and genome organization
R-loops are three-stranded nucleic acid structures that are abundant and widespread across
the genome and that have important physiological roles in many nuclear processes. Their …
the genome and that have important physiological roles in many nuclear processes. Their …
In vivo prime editing of a metabolic liver disease in mice
Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …
without DNA double-strand break formation. Despite rapid technological advances, in vivo …
Efficient targeted insertion of large DNA fragments without DNA donors
J Wang, Z He, G Wang, R Zhang, J Duan, P Gao… - Nature …, 2022 - nature.com
Targeted insertion of large DNA fragments holds great potential for treating genetic
diseases. Prime editors can effectively insert short fragments (~ 44 bp) but not large ones …
diseases. Prime editors can effectively insert short fragments (~ 44 bp) but not large ones …