[HTML][HTML] 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 …

[HTML][HTML] Small extracellular vesicles (sEVs)-based gene delivery platform for cell-specific CRISPR/Cas9 genome editing

S Dubey, Z Chen, YJ Jiang, A Talis, A Molotkov… - Theranostics, 2024 - ncbi.nlm.nih.gov
Small extracellular vesicles (sEVs) are naturally occurring vesicles that have the potential to
be manipulated to become promising drug delivery vehicles for on-demand in vitro and 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 …

[HTML][HTML] 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 …

[HTML][HTML] Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets

M Taghdiri, C Mussolino - International Journal of Molecular Sciences, 2024 - mdpi.com
Clustered regularly interspersed short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) technology has revolutionized the field of gene therapy as it has enabled …

Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation

W Zhu, W Du, AP Rameshbabu… - Science translational …, 2024 - science.org
Mutations in microRNA-96 (MIR96) cause autosomal dominant deafness-50 (DFNA50), a
form of delayed-onset hearing loss. Genome editing has shown efficacy in hearing recovery …

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 …

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 …