Making Gene Editing a Therapeutic Reality

Overview

Journal F1000Res

Specialties Biomedical Engineering
Science

Date 2019 Jan 8

PMID 30613384

Citations 9

Authors

Irina Conboy

Niren Murthy

Jessy Etienne

Zachery Robinson

Affiliations

Soon will be listed here.

Abstract

This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.

Citing Articles

Delivery of CRISPR/Cas9 Plasmid DNA by Hyperbranched Polymeric Nanoparticles Enables Efficient Gene Editing.

Xiu K, Saunders L, Wen L, Ruan J, Dong R, Song J Cells. 2023; 12(1).

PMID: 36611948 PMC: 9818138. DOI: 10.3390/cells12010156.

Novel vectors and approaches for gene therapy in liver diseases.

Maestro S, Weber N, Zabaleta N, Aldabe R, Gonzalez-Aseguinolaza G JHEP Rep. 2021; 3(4):100300.

PMID: 34159305 PMC: 8203845. DOI: 10.1016/j.jhepr.2021.100300.

A novel chemical-combination screen in zebrafish identifies epigenetic small molecule candidates for the treatment of Duchenne muscular dystrophy.

Farr 3rd G, Morris M, Gomez A, Pham T, Kilroy E, Parker E Skelet Muscle. 2020; 10(1):29.

PMID: 33059738 PMC: 7559456. DOI: 10.1186/s13395-020-00251-4.

Effective restoration of dystrophin expression in iPSC -derived muscle progenitor cells using the CRISPR/Cas9 system and homology-directed repair technology.

Jin Y, Shen Y, Su X, Weintraub N, Tang Y Comput Struct Biotechnol J. 2020; 18:765-773.

PMID: 32280431 PMC: 7132053. DOI: 10.1016/j.csbj.2020.03.012.

Is Alzheimer's Disease a Liver Disease of the Brain?.

Bassendine M, Taylor-Robinson S, Fertleman M, Khan M, Neely D J Alzheimers Dis. 2020; 75(1):1-14.

PMID: 32250293 PMC: 7306895. DOI: 10.3233/JAD-190848.

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