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 …

[HTML][HTML] Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

S Zhang, J Shen, D Li, Y Cheng - Theranostics, 2021 - ncbi.nlm.nih.gov
CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag

SQ Liang, P Liu, K Ponnienselvan, S Suresh, Z Chen… - Nature …, 2023 - nature.com
Prime editors have a broad range of potential research and clinical applications. However,
methods to delineate their genome-wide editing activities have generally relied on indirect …

Optimization of AsCas12a for combinatorial genetic screens in human cells

PC DeWeirdt, KR Sanson, AK Sangree, M Hegde… - Nature …, 2021 - nature.com
Cas12a RNA-guided endonucleases are promising tools for multiplexed genetic
perturbations because they can process multiple guide RNAs expressed as a single …

AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines

L Zhang, JA Zuris, R Viswanathan, JN Edelstein… - Nature …, 2021 - nature.com
Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits
relatively poor editing efficiency, restricting its overall utility. Here we isolate an engineered …

Multiplexed genome regulation in vivo with hyper-efficient Cas12a

LY Guo, J Bian, AE Davis, P Liu, HR Kempton… - Nature cell …, 2022 - nature.com
Multiplexed modulation of endogenous genes is crucial for sophisticated gene therapy and
cell engineering. CRISPR–Cas12a systems enable versatile multiple-genomic-loci targeting …

Zebrafish models of cancer—new insights on modeling human cancer in a non-mammalian vertebrate

M Hason, P Bartůněk - Genes, 2019 - mdpi.com
Zebrafish (Danio rerio) is a valuable non-mammalian vertebrate model widely used to study
development and disease, including more recently cancer. The evolutionary conservation of …

New CRISPR-derived microRNA sensing mechanism based on Cas12a self-powered and rolling circle transcription-unleashed real-time crRNA recruiting

G Wang, W Tian, X Liu, W Ren, C Liu - Analytical chemistry, 2020 - ACS Publications
Current CRISPR-Cas-based nucleic acid sensing methods relying on the preassembled
Cas-crRNA complexes are generally limited to the detection of protospacer-adjacent motif …

Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

SQ Liang, P Liu, JL Smith, E Mintzer, S Maitland… - Nature …, 2022 - nature.com
Abstract Analysis of off-target editing is an important aspect of the development of safe
nuclease-based genome editing therapeutics. in vivo assessment of nuclease off-target …

Precise therapeutic gene correction by a simple nuclease-induced double-stranded break

S Iyer, S Suresh, D Guo, K Daman, JCJ Chen, P Liu… - Nature, 2019 - nature.com
Current programmable nuclease-based methods (for example, CRISPR–Cas9) for the
precise correction of a disease-causing genetic mutation harness the homology-directed …