Article

Dual-gRNA Approach With Limited Off-Target Effect Corrects C9ORF72 Repeat Expansion In Vivo

2021-09-23

Abstract excerpt

C9ORF72 GGGGCC repeat expansion is the most common genetic cause for amyotrophic lateral sclerosis and frontotemporal dementia, which generates abnormal DNA and RNA structures and produces toxic proteins. Recently, efficacy of CRISPR/Cas9-mediated editing has been proven in treatment of disease. However, DNA low complexity surrounding C9ORF72 expansion increases the off-target risks. Here we provide a dual-gRNA de...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 66e11408-359f-503a-ac33-83b9cb223f7e
DOI: 10.21203/rs.3.rs-870138/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Dual-gRNA Approach With Limited Off-Target Effect Corrects C9ORF72 Repeat Expansion In VivoDOI 10.21203/rs.3.rs-870138/v1Read

A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTDSemantic proximity Read

A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTDSemantic proximity Read

Two therapeutic CRISPR/Cas9 gene editing approaches revert FTD/ALS cellular pathology caused by a <i>C9orf72</i> repeat expansion mutation in patient derived cellsSemantic proximity Read

CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitroSemantic proximity Read

CRISPR-Cas9 targeted deletion of the <i>C9orf72</i> repeat expansion mutation corrects cellular phenotypes in patient-derived iPS cellsSemantic proximity Read

Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivoSemantic proximity Read

CRISPR/Cas9-Mediated Excision of ALS/FTD-Causing Hexanucleotide Repeat Expansion in <i>C9ORF72</i> rescues major disease mechanisms <i>in vivo</i> and <i>in vitro</i>Semantic proximity Read

Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene TherapySemantic proximity Read

The Emerging Role of DNA Damage in the Pathogenesis of the C9orf72 Repeat Expansion in Amyotrophic Lateral SclerosisSemantic proximity Read

Dual-targeting CRISPR-CasRx reduces <i>C9orf72</i> ALS/FTD sense and antisense repeat RNAs in vitro and in vivoSemantic proximity Read

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.Semantic proximity Read

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAsSemantic proximity Read