Abstract
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 nuclease (Cas9) is a genome editing tool that has recently caught enormous attention due to its novelty, feasibility, and affordability. This system naturally functions as a defense mechanism in bacteria and has been repurposed as an RNA-guided DNA editing tool. Unlike zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), CRISPR/Cas9 takes advantage of an RNA-guided DNA endonuclease enzyme, Cas9, which is able to generate double-strand breaks (DSBs) at specific genomic locations. It triggers cellular endogenous DNA repair pathways, contributing to the generation of desired modifications in the genome. The ability of the system to precisely disrupt DNA sequences has opened up new avenues in our understanding of amyotrophic lateral sclerosis (ALS) pathogenesis and the development of new therapeutic approaches. In this review, we discuss the current knowledge of the principles and limitations of the CRISPR/Cas9 system, as well as strategies to improve these limitations. Furthermore, we summarize novel approaches of engaging the CRISPR/Cas9 system in establishing an adequate model of neurodegenerative disease and in the treatment of SOD1-linked forms of ALS. We also highlight possible applications of this system in the therapy of ALS, both the inherited type as well as ALS of sporadic origin.
Highlights
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 nuclease (Cas9) is a novel genome editing tool that has recently revolutionized the field of human genetic engineering
The results of this study have revealed the enormous potential of the CRISPR/Cas9 system in the modification of multiple genes in pigs and the results have confirmed the utility of the system in modeling neurodegenerative diseases
The CRISPR/Cas9 system makes genome engineering technology feasible for application in many fields including human diseases. Current research on this innovative genome editing tool and strategies to improve its drawbacks has laid the groundwork for future clinical work in neurodegenerative diseases
Summary
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 nuclease (Cas9) is a novel genome editing tool that has recently revolutionized the field of human genetic engineering. LLiimmiittaattiioonnss ooff tthhee CCRRIISSPPRR//CCaass99 SSyysstteemm TThhee ssimimpplilcicitiytyofotfhethCeRCISRPIRS/PCRa/Cs9ass9ystseymsteemncoeunrcaoguersatgheesexthpeloreaxtpiolnoroaftiiotsnpooftenittsiapl tootegnetniaelrattoe ganenimeraaltemaondimelsalomr toodterlesaotrsetovetrreeaatnsdevinerceuraanbdleinhcuumraabnledhisuemasaens.dHiseoawseesv.eHr, opwoteevnetri,apl uotseenrstianleuedsetros ncoenedsidtoercosenvseidraelrissesvueersatlhiasstuliems itthtahtelifmeaistitbhielitfyeaosfibthiliistysyosfttehmis. The application of the CRISPR/Cas9 system as a novel tool for modeling neurodegenerative diseases has been facilitated due to its ability to generate mutations in endogenous genes.
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