CRISPR technologies and the search for the PAM-free nuclease

D Collias, CL Beisel - Nature communications, 2021 - nature.com
The ever-expanding set of CRISPR technologies and their programmable RNA-guided
nucleases exhibit remarkable flexibility in DNA targeting. However, this flexibility comes with …

CRISPR-Cas9 DNA base-editing and prime-editing

A Kantor, ME McClements, RE MacLaren - International journal of …, 2020 - mdpi.com
Many genetic diseases and undesirable traits are due to base-pair alterations in genomic
DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic …

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

ML Leibowitz, S Papathanasiou, PA Doerfler… - Nature …, 2021 - nature.com
Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …

The CRISPR tool kit for genome editing and beyond

M Adli - Nature communications, 2018 - nature.com
CRISPR is becoming an indispensable tool in biological research. Once known as the
bacterial immune system against invading viruses, the programmable capacity of the Cas9 …

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

ML Maeder, M Stefanidakis, CJ Wilson, R Baral… - Nature medicine, 2019 - nature.com
Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the
CEP290 gene,. We developed EDIT-101, a candidate genome-editing therapeutic, to …

The emerging and uncultivated potential of CRISPR technology in plant science

Y Zhang, AA Malzahn, S Sretenovic, Y Qi - Nature Plants, 2019 - nature.com
The application of clustered regularly interspaced short palindromic repeats (CRISPR) for
genetic manipulation has revolutionized life science over the past few years. CRISPR was …

[HTML][HTML] In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

M Behr, J Zhou, B Xu, H Zhang - Acta Pharmaceutica Sinica B, 2021 - Elsevier
Within less than a decade since its inception, CRISPR-Cas9-based genome editing has
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …

CRISPR-based technologies for the manipulation of eukaryotic genomes

AC Komor, AH Badran, DR Liu - Cell, 2017 - cell.com
The CRISPR-Cas9 RNA-guided DNA endonuclease has contributed to an explosion of
advances in the life sciences that have grown from the ability to edit genomes within living …

Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

C Liu, L Zhang, H Liu, K Cheng - Journal of controlled release, 2017 - Elsevier
The CRISPR-Cas9 genome-editing system is a part of the adaptive immune system in
archaea and bacteria to defend against invasive nucleic acids from phages and plasmids …

CRISPR/Cas9-based genome editing for disease modeling and therapy: challenges and opportunities for nonviral delivery

HX Wang, M Li, CM Lee, S Chakraborty… - Chemical …, 2017 - ACS Publications
Genome editing offers promising solutions to genetic disorders by editing DNA sequences
or modulating gene expression. The clustered regularly interspaced short palindromic …