Therapeutic genome editing: prospects and challenges

DBT Cox, RJ Platt, F Zhang - Nature medicine, 2015 - nature.com
Recent advances in the development of genome editing technologies based on
programmable nucleases have substantially improved our ability to make precise changes …

Genome editing: the road of CRISPR/Cas9 from bench to clinic

A Eid, MM Mahfouz - Experimental & Molecular Medicine, 2016 - nature.com
Molecular scissors engineered for site-specific modification of the genome hold great
promise for effective functional analyses of genes, genomes and epigenomes and could …

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

M Kosicki, K Tomberg, A Bradley - Nature biotechnology, 2018 - nature.com
CRISPR–Cas9 is poised to become the gene editing tool of choice in clinical contexts. Thus
far, exploration of Cas9-induced genetic alterations has been limited to the immediate …

DNA-free genetically edited grapevine and apple protoplast using CRISPR/Cas9 ribonucleoproteins

M Malnoy, R Viola, MH Jung, OJ Koo, S Kim… - Frontiers in plant …, 2016 - frontiersin.org
The combined availability of whole genome sequences and genome editing tools is set to
revolutionize the field of fruit biotechnology by enabling the introduction of targeted genetic …

DNA repair profiling reveals nonrandom outcomes at Cas9-mediated breaks

M van Overbeek, D Capurso, MM Carter… - Molecular cell, 2016 - cell.com
The repair outcomes at site-specific DNA double-strand breaks (DSBs) generated by the
RNA-guided DNA endonuclease Cas9 determine how gene function is altered. Despite the …

Real-time observation of DNA recognition and rejection by the RNA-guided endonuclease Cas9

D Singh, SH Sternberg, J Fei, JA Doudna… - Nature communications, 2016 - nature.com
Binding specificity of Cas9–guide RNA complexes to DNA is important for genome-
engineering applications; however, how mismatches influence target recognition/rejection …

CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sites

R Jayavaradhan, DM Pillis, M Goodman… - Nature …, 2019 - nature.com
Precise genome editing/correction of DNA double-strand breaks (DSBs) induced by
CRISPR-Cas9 by homology-dependent repair (HDR) is limited by the competing error-prone …

Microhomologies are prevalent at Cas9-induced larger deletions

DDG Owens, A Caulder, V Frontera… - Nucleic acids …, 2019 - academic.oup.com
The CRISPR system is widely used in genome editing for biomedical research. Here, using
either dual paired Cas9D10A nickases or paired Cas9 nuclease we characterize …

Cas9-induced large deletions and small indels are controlled in a convergent fashion

M Kosicki, F Allen, F Steward, K Tomberg, Y Pan… - Nature …, 2022 - nature.com
Repair of Cas9-induced double-stranded breaks results primarily in formation of small
insertions and deletions (indels), but can also cause potentially harmful large deletions …

An episomal vector-based CRISPR/Cas9 system for highly efficient gene knockout in human pluripotent stem cells

Y Xie, D Wang, F Lan, G Wei, T Ni, R Chai, D Liu… - Scientific reports, 2017 - nature.com
Human pluripotent stem cells (hPSCs) represent a unique opportunity for understanding the
molecular mechanisms underlying complex traits and diseases. CRISPR/Cas9 is a powerful …