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 …

CRISPR/Cas gene therapy

B Zhang - Journal of cellular physiology, 2021 - Wiley Online Library
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated
enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic …

High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition

S Selvaraj, WN Feist, S Viel, S Vaidyanathan… - Nature …, 2024 - nature.com
Therapeutic applications of nuclease-based genome editing would benefit from improved
methods for transgene integration via homology-directed repair (HDR). To improve HDR …

Saturation variant interpretation using CRISPR prime editing

S Erwood, TMI Bily, J Lequyer, J Yan, N Gulati… - Nature …, 2022 - nature.com
High-throughput functional characterization of genetic variants in their endogenous locus
has so far been possible only with methods that rely on homology-directed repair, which are …

Genome-wide CRISPR off-target prediction and optimization using RNA-DNA interaction fingerprints

Q Chen, G Chuai, H Zhang, J Tang, L Duan… - Nature …, 2023 - nature.com
The powerful CRISPR genome editing system is hindered by its off-target effects, and
existing computational tools achieved limited performance in genome-wide off-target …

Base editing screens map mutations affecting interferon-γ signaling in cancer

MA Coelho, S Cooper, ME Strauss, E Karakoc… - Cancer Cell, 2023 - cell.com
Summary Interferon-γ (IFN-γ) signaling mediates host responses to infection, inflammation
and anti-tumor immunity. Mutations in the IFN-γ signaling pathway cause immunological …

CRISPR/dCas9 tools: epigenetic mechanism and application in gene transcriptional regulation

R Cai, R Lv, X Shi, G Yang, J ** - International journal of molecular …, 2023 - mdpi.com
CRISPR/Cas9-mediated cleavage of DNA, which depends on the endonuclease activity of
Cas9, has been widely used for gene editing due to its excellent programmability and …

Improvements in gene editing technology boost its applications in livestock

IV Perisse, Z Fan, GN Singina, KL White… - Frontiers in …, 2021 - frontiersin.org
Accelerated development of novel CRISPR/Cas9-based genome editing techniques
provides a feasible approach to introduce a variety of precise modifications in the …

[PDF][PDF] Gene therapy and gene editing for β-thalassemia

GE Christakopoulos, R Telange, J Yen… - … /oncology clinics of North …, 2023 - Elsevier
After many years of painstaking research, the potential of gene therapy to cure severe β-
thalassemia is now becoming evident through recent clinical trials. Therapeutic protocols …

A potential paradigm in CRISPR/Cas systems delivery: at the crossroad of microalgal gene editing and algal-mediated nanoparticles

S Feng, X **e, J Liu, A Li, Q Wang, D Guo, S Li… - Journal of …, 2023 - Springer
Microalgae as the photosynthetic organisms offer enormous promise in a variety of
industries, such as the generation of high-value byproducts, biofuels, pharmaceuticals …