Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination Using CRISPR/Cas9 System

Overview

Journal PLoS Curr

Specialty General Medicine

Date 2014 Apr 25

PMID 24761311

Citations 31

Authors

Mahru C An

Robert N OBrien

Ningzhe Zhang

Biranchi N Patra

Michael De La Cruz

Animesh Ray

Lisa M Ellerby

Affiliations

Soon will be listed here.

Abstract

We have previously reported the genetic correction of Huntington's disease (HD) patient-derived induced pluripotent stem cells using traditional homologous recombination (HR) approaches. To extend this work, we have adopted a CRISPR-based genome editing approach to improve the efficiency of recombination in order to generate allelic isogenic HD models in human cells. Incorporation of a rapid antibody-based screening approach to measure recombination provides a powerful method to determine relative efficiency of genome editing for modeling polyglutamine diseases or understanding factors that modulate CRISPR/Cas9 HR.

Citing Articles

CRISPR/Cas9-mediated genetic correction reverses spinocerebellar ataxia 3 disease-associated phenotypes in differentiated cerebellar neurons.

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Therapeutic approaches targeting aging and cellular senescence in Huntington's disease.

Bhat A, Moglad E, Afzal M, Thapa R, Almalki W, Kazmi I CNS Neurosci Ther. 2024; 30(10):e70053.

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CRISPR-Based Genome-Editing Tools for Huntington's Disease Research and Therapy.

Qin Y, Li S, Li X, Yang S Neurosci Bull. 2022; 38(11):1397-1408.

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Advances in Modeling Polyglutamine Diseases Using Genome Editing Tools.

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