[HTML][HTML] Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022 - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

[PDF][PDF] The CRISPR-Cas toolbox and gene editing technologies

G Liu, Q Lin, S Jin, C Gao - Molecular cell, 2022 - cell.com
The emergence of CRISPR-Cas systems has accelerated the development of gene editing
technologies, which are widely used in the life sciences. To improve the performance of …

Targeted DNA integration in human cells without double-strand breaks using CRISPR-associated transposases

GD Lampe, RT King, TS Halpin-Healy… - Nature …, 2024 - nature.com
Conventional genome engineering with CRISPR–Cas9 creates double-strand breaks
(DSBs) that lead to undesirable byproducts and reduce product purity. Here we report an …

High-fidelity Cas13 variants for targeted RNA degradation with minimal collateral effects

H Tong, J Huang, Q Xiao, B He, X Dong, Y Liu… - Nature …, 2023 - nature.com
CRISPR–Cas13 systems have recently been used for targeted RNA degradation in various
organisms. However, collateral degradation of bystander RNAs has limited their in vivo …

[PDF][PDF] Structure and engineering of the type III-E CRISPR-Cas7-11 effector complex

K Kato, W Zhou, S Okazaki, Y Isayama, T Nishizawa… - Cell, 2022 - cell.com
Summary The type III-E CRISPR-Cas effector Cas7-11, with dual RNase activities for
precursor CRISPR RNA (pre-crRNA) processing and crRNA-guided target RNA cleavage, is …

RNA-activated protein cleavage with a CRISPR-associated endopeptidase

J Strecker, FE Demircioglu, D Li, G Faure… - Science, 2022 - science.org
In prokaryotes, CRISPR-Cas systems provide adaptive immune responses against foreign
genetic elements through RNA-guided nuclease activity. Recently, additional genes with …

Big data mining, rational modification, and ancestral sequence reconstruction inferred multiple xylose isomerases for biorefinery

S Chen, Z Xu, B Ding, Y Zhang, S Liu, C Cai, M Li… - Science …, 2023 - science.org
The isomerization of xylose to xylulose is considered the most promising approach to initiate
xylose bioconversion. Here, phylogeny-guided big data mining, rational modification, and …

CRISPR/Cas13 effectors have differing extents of off-target effects that limit their utility in eukaryotic cells

Y Ai, D Liang, JE Wilusz - Nucleic Acids Research, 2022 - academic.oup.com
CRISPR/Cas13 effectors have garnered increasing attention as easily customizable tools for
detecting and depleting RNAs of interest. Near perfect complementarity between a target …

Precise transcript targeting by CRISPR-Csm complexes

D Colognori, M Trinidad, JA Doudna - Nature biotechnology, 2023 - nature.com
Robust and precise transcript targeting in mammalian cells remains a difficult challenge
using existing approaches due to inefficiency, imprecision and subcellular …

Craspase is a CRISPR RNA-guided, RNA-activated protease

C Hu, SPB van Beljouw, KH Nam, G Schuler, F Ding… - Science, 2022 - science.org
The CRISPR-Cas type III-E RNA-targeting effector complex gRAMP/Cas7-11 is associated
with a caspase-like protein (TPR-CHAT/Csx29) to form Craspase (CRISPR-guided …