Efficient high-precision homology-directed repair-dependent genome editing by HDRobust

S Riesenberg, P Kanis, D Macak, D Wollny… - Nature …, 2023 - nature.com
Abstract Homology-directed repair (HDR), a method for repair of DNA double-stranded
breaks can be leveraged for the precise introduction of mutations supplied by synthetic DNA …

CRISPR/Cas9 landscape: Current state and future perspectives

M Tyumentseva, A Tyumentsev, V Akimkin - International Journal of …, 2023 - mdpi.com
CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 is a unique
genome editing tool that can be easily used in a wide range of applications, including …

Enhancing homology-directed repair efficiency with HDR-boosting modular ssDNA donor

YY Jin, P Zhang, LL Liu, X Zhao, XQ Hu, SZ Liu… - Nature …, 2024 - nature.com
Despite the potential of small molecules and recombinant proteins to enhance the efficiency
of homology-directed repair (HDR), single-stranded DNA (ssDNA) donors, as currently …

CRISPR‐Cas9 screening identified lethal genes enriched in necroptosis pathway and of prognosis significance in osteosarcoma

L Cheng, W Xiong, S Li, G Wang… - The Journal of Gene …, 2023 - Wiley Online Library
Background The present study aimed to identify indispensable genes associated with tumor
cell viability according to the clustered regularly interspaced short palindromic repeats …

Improving homology‐directed repair by small molecule agents for genetic engineering in unconventional yeast?—Learning from the engineering of mammalian …

M Lu, S Billerbeck - Microbial Biotechnology, 2024 - Wiley Online Library
The ability to precisely edit genomes by deleting or adding genetic information enables the
study of biological functions and the building of efficient cell factories. In many …

Small molecule mif modulation enhances ferroptosis by impairing DNA repair mechanisms

D Chen, C Zhao, J Zhang, CWJ Knol… - Advanced …, 2024 - Wiley Online Library
Ferroptosis is a form of regulated cell death that can be modulated by small molecules and
has the potential for the development of therapeutics for oncology. Although excessive lipid …

Weaving the gates of life: Pioneering a new era in oral gene delivery with metal-organic frameworks

M Jiang, G Zhang, Q Zeng, D Xiong, X Bai, Y Wu… - Chemical Engineering …, 2024 - Elsevier
Gene therapy has transformed personalized medicine by enabling tailored treatments based
on genetic profiles. While traditional delivery methods like intramuscular injections are …

Identification of a Cryptic Binding Site in CRISPR-Cas9 for Targeted Inhibition

N Zhang, Z Zuo - Journal of Chemical Information and Modeling, 2023 - ACS Publications
The need for precision control of CRISPR-Cas9 genome editing has created a demand for
anti-CRISPR molecules. Recently, the first class of small-molecule Cas9 inhibitors has been …

Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy

S Hasselbeck, X Cheng - Pharmaceuticals, 2023 - mdpi.com
In the rapidly evolving landscape of genetic engineering, the advent of CRISPR-Cas
technologies has catalyzed a paradigm shift, empowering scientists to manipulate the …

In situ correction of various β-thalassemia mutations in human hematopoietic stem cells

Y Yang, L He, Y Xie, L Zhu, J Wu, Y Fan… - Frontiers in Cell and …, 2024 - frontiersin.org
β-thalassemia (β-thal) is the most common monogenic disorder caused by various mutations
in the human hemoglobin β (HBB) gene and affecting millions of people worldwide …