A novel genome-wide CRISPR screening strategy that will facilitate the systematic engineering of novel nonviral genome editing delivery methods, where the identified novel gene hits can be further used to increase editing efficiency for other therapeutically relevant cell types.
To systematically map cellular factors constraining nonviral genome editing, influencing uptake and intracellular trafficking, we develop a genome-wide CRISPR screening platform linking perturbation of 19,114 genes to editing outcomes in human cells. We identify six negative regulators of delivery whose depletion incre...
Shivani Saxena, Meha Kabra, Amr A. Abdeen et al.· Nature Communications· 0 citations
A comprehensive review of delivery modalities of CRISPR systems solely in vivo that underpin their therapeutic translation and outlines the remaining barriers to durable, tissue-selective, and broadly deployable CRISPR therapeutics is provided.
L. Martin, Jure Bohinc, Alessandra Recchia et al.· International Journal on Bio...· 0 citations
Cells in multicellular eukaryotic systems are diverse biological units, with characteristics and functions determined by their molecular profiles. CRISPR–Cas9 genome editing has been widely used across biology to modulate gene expression and study gene function. However, there is currently no versatile and scalable met...
Arman Adel, Yuta Shuto, Shunsuke Kawasaki et al.· bioRxiv· 0 citations
CRISPR editors including nucleases, base editors, and prime editors can efficiently correct disease-causing genetic variants or disrupt target genes. Editing outcomes are commonly evaluated in cultured primary cells, patient-derived cells, or engineered cell lines to study the impact of genetic variation or as a first...
Maria Viskadourou, J. Workman, Emma V Burke et al.· Journal of Visualized Experi...· 0 citations
The types, principles and characteristics of gene editing systems are introduced in order to understand their requirements for delivery tools and to provide new insights to facilitate appropriate delivery systems or improve the efficacy of existing systems.
Fanyu Xue, Zixuan Xin, Gaojie Wang et al.· Artificial Cells Nanomedicin...· 0 citations
Genomic manipulation has advanced from stochastic nuclease‐mediated disruption toward programmable, deterministic precision. Early clustered regularly interspaced short palindromic repeats (CRISPR) strategies enabled targeted mutagenesis through double‐strand breaks; however, their therapeutic application is limited by...