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 …

Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing

J Tao, DE Bauer, R Chiarle - Nature Communications, 2023 - nature.com
CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells

CA Tsuchida, N Brandes, R Bueno, M Trinidad… - Cell, 2023 - cell.com
CRISPR-Cas9 genome editing has enabled advanced T cell therapies, but occasional loss
of the targeted chromosome remains a safety concern. To investigate whether Cas9-induced …

Development of miniature base editors using engineered IscB nickase

D Han, Q Xiao, Y Wang, H Zhang, X Dong, G Li… - Nature …, 2023 - nature.com
As a miniature RNA-guided endonuclease, IscB is presumed to be the ancestor of Cas9 and
to share similar functions. IscB is less than half the size of Cas9 and thus more suitable for in …

Unintended CRISPR-Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene-editing in human cells

JMT Hunt, CA Samson, A Rand, HM Sheppard - Human Genetics, 2023 - Springer
Genome editing using the clustered regularly interspaced short palindromic repeats
(CRISPR) and CRISPR-associated protein (Cas) gene-editing system (CRISPR-Cas) is a …

Metabolic engineering of plant secondary metabolites: prospects and its technological challenges

A Mipeshwaree Devi, K Khedashwori Devi… - Frontiers in Plant …, 2023 - frontiersin.org
Plants produce a wide range of secondary metabolites that play vital roles for their primary
functions such as growth, defence, adaptations or reproduction. Some of the plant …

CRISPRthripsis: The risk of CRISPR/Cas9-induced chromothripsis in gene therapy

M Amendola, M Brusson, A Miccio - Stem Cells Translational …, 2022 - academic.oup.com
Abstract The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9
nuclease system has allowed the generation of disease models and the development of …

Combining different CRISPR nucleases for simultaneous knock-in and base editing prevents translocations in multiplex-edited CAR T cells

V Glaser, C Flugel, J Kath, W Du, V Drosdek, C Franke… - Genome Biology, 2023 - Springer
Background Multiple genetic modifications may be required to develop potent off-the-shelf
chimeric antigen receptor (CAR) T cell therapies. Conventional CRISPR-Cas nucleases …

CRISPR nuclease off-target activity and mitigation strategies

B Wienert, MK Cromer - Frontiers in Genome Editing, 2022 - frontiersin.org
The discovery of CRISPR has allowed site-specific genomic modification to become a reality
and this technology is now being applied in a number of human clinical trials. While this …

Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration

J Yin, K Fang, Y Gao, L Ou, S Yuan, C Xin… - Nature …, 2022 - nature.com
Ensuring genome safety during gene editing is crucial for clinical translation of the high-
efficient CRISPR-Cas9 toolbox. Therefore, the undesired events including chromosomal …