Specific knockdown of Htra2 by CRISPR-CasRx prevents acquired sensorineural hearing loss in mice

Y Guo, L Han, S Han, H Tang, S Wang, C Cui… - … Therapy-Nucleic Acids, 2022 - cell.com
CasRx, a recently discovered member of the type VI CRISPR system with minimum size,
offers a new approach for RNA manipulation with high efficiency and specificity in …

Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins

JM Metzger, Y Wang, SS Neuman, KJ Snow… - Biomaterials, 2023 - Elsevier
Genome editing of somatic cells via clustered regularly interspaced short palindromic
repeats (CRISPR) offers promise for new therapeutics to treat a variety of genetic disorders …

Preparation of NanoMEDIC Extracellular Vesicles to Deliver CRISPR-Cas9 Ribonucleoproteins for Genomic Exon Skipping

K Watanabe, P Gee, A Hotta - Muscular Dystrophy Therapeutics: Methods …, 2022 - Springer
The CRISPR-Cas9 system has quickly become the standard tool for genome editing. To
deliver this system to target cells, adeno-associated virus (AAV) vectors are commonly used …

A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing

G Chen, AA Abdeen, Y Wang, PK Shahi… - Nature …, 2019 - nature.com
Delivery technologies for the CRISPR-Cas9 (CRISPR, clustered regularly interspaced short
palindromic repeats) gene editing system often require viral vectors, which pose safety …

Engineered extracellular vesicle-delivered CRISPR/Cas9 for radiotherapy sensitization of glioblastoma

X Liu, Z Cao, W Wang, C Zou, Y Wang, L Pan, B Jia… - ACS …, 2023 - ACS Publications
Radiotherapy is a mainstay of glioblastoma (GBM) treatment; however, the development of
therapeutic resistance has hampered the efficacy of radiotherapy, suggesting that additional …

Preventing autosomal-dominant hearing loss in Bth mice with CRISPR/CasRx-based RNA editing

Z Zheng, G Li, C Cui, F Wang, X Wang, Z Xu… - … and Targeted Therapy, 2022 - nature.com
Abstract CRISPR/RfxCas13d (CasRx) editing system can specifically and precisely cleave
single-strand RNAs, which is a promising treatment for various disorders by downregulation …

Brain-targeted CRISPR/Cas9 nanomedicine for effective glioblastoma therapy

W Ruan, M Jiao, S Xu, M Ismail, X Xie, Y An… - Journal of Controlled …, 2022 - Elsevier
CRISPR/Cas9 gene-editing technology shows great potential for treating a variety of
diseases, such as glioblastoma multiforme (GBM). However, CRISPR components suffer …

An optimized SpCas9 high-fidelity variant for direct protein delivery

E Pedrazzoli, A Bianchi, A Umbach, S Amistadi… - Molecular Therapy, 2023 - cell.com
Electroporation of the Cas9 ribonucleoprotein (RNP) complex offers the advantage of
preventing off-target cleavages and potential immune responses produced by long-term …

Targeted Therapeutic Genome Engineering: Opportunities and Bottlenecks in Medical Translation

JA Kretzmann, DC Luther, M Norret… - Targeted …, 2019 - ACS Publications
Tools for editing the genome and epigenome represent a new frontier in targeted
therapeutic intervention. Nonviral nanoparticle-based delivery systems are potentially the …

High-throughput in situ cell electroporation microsystem for parallel delivery of single guide RNAs into mammalian cells

S Bian, Y Zhou, Y Hu, J Cheng, X Chen, Y Xu, P Liu - Scientific reports, 2017 - nature.com
Arrayed genetic screens mediated by the CRISPR/Cas9 technology with single guide RNA
(sgRNA) libraries demand a high-throughput platform capable of transfecting diverse cell …