CRISPR–Cas-related technologies in basic and translational liver research

Song, Chun‐Qing; Xue, Wen

doi:10.1038/nrgastro.2018.11

Cited by 10 publications

(9 citation statements)

References 10 publications

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“…Cas13 differs from Cas9 because binds and cleaves RNA instead of DNA, which could be a promising tool for RNA-directed therapies [186]. Several preclinical studies used CRISPR effectors to edit DNA or RNA but efficient translation into clinical practice remains to be verified [187,188]. Small molecules .…”

Section: Lncrnas Diagnostic Prognostic and Therapeutic Perspectivesmentioning

confidence: 99%

Long Noncoding RNA and Epithelial Mesenchymal Transition in Cancer

Gugnoni

Ciarrocchi

2019

IJMS

123

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Epithelial–mesenchymal transition (EMT) is a multistep process that allows epithelial cells to acquire mesenchymal properties. Fundamental in the early stages of embryonic development, this process is aberrantly activated in aggressive cancerous cells to gain motility and invasion capacity, thus promoting metastatic phenotypes. For this reason, EMT is a central topic in cancer research and its regulation by a plethora of mechanisms has been reported. Recently, genomic sequencing and functional genomic studies deepened our knowledge on the fundamental regulatory role of noncoding DNA. A large part of the genome is transcribed in an impressive number of noncoding RNAs. Among these, long noncoding RNAs (lncRNAs) have been reported to control several biological processes affecting gene expression at multiple levels from transcription to protein localization and stability. Up to now, more than 8000 lncRNAs were discovered as selectively expressed in cancer cells. Their elevated number and high expression specificity candidate these molecules as a valuable source of biomarkers and potential therapeutic targets. Rising evidence currently highlights a relevant function of lncRNAs on EMT regulation defining a new layer of involvement of these molecules in cancer biology. In this review we aim to summarize the findings on the role of lncRNAs on EMT regulation and to discuss their prospective potential value as biomarkers and therapeutic targets in cancer.

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Section: Lncrnas Diagnostic Prognostic and Therapeutic Perspectivesmentioning

confidence: 99%

Long Noncoding RNA and Epithelial Mesenchymal Transition in Cancer

Gugnoni

Ciarrocchi

2019

IJMS

123

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“…We and others recently reported that CRISPR can correct this Fah mutation through HDR [16][17][18] or allelic exchange 19 . Following correction by CRISPR, liver cells expressing the Fah enzyme, through their selective advantage, expand and repopulate the liver 14 .…”

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confidence: 95%

Adenine base editing in an adult mouse model of tyrosinaemia

et al. 2019

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Unlike traditional CRISPR-Cas9 homology-directed repair, base editing can correct point mutations without supplying a DNA-repair template. Here, we show in a mouse model of tyrosinemia that hydrodynamic tail-vein injection of plasmid DNA encoding the adenine base editor (ABE) and a single guide RNA can correct an A>G splice-site mutation. ABE treatment partially restored splicing, generated fumarylacetoacetate hydrolase (Fah)-positive hepatocytes in the liver, and rescued weight loss in the animals. We also generated Fah+ hepatocytes in the liver via lipid-nanoparticle-mediated delivery of chemically modified sgRNA and an mRNA of a codon-optimized base editor that displayed higher base-editing efficiency than the standard ABE. Our findings suggest that adenosine base editing can be used for the correction of genetic disease in adult animals.

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“…The working mechanism of the CRISPR/Cas system differs from other genome editing platforms, for it uses an RNA molecule more than a protein to recognize DNA [25]. CRISPR/Cas systems are classified into three types (I-III).…”

Section: Overview Of Crispr/cas9 Techniquementioning

confidence: 99%

“…Additionally, the genome editing efficiency is also affected by non-homologous end joining (NHEJ) and homology-directed repair (HDR) DNA repair mechanisms [4]. Studies have demonstrated that the HDR rate is lower than the NHEJ rate in the liver, limiting the therapy of diseases requiring gene editing by HDR [25]. Thankfully, the latest research has revealed the Fanconi anemia pathway to make CRISPR/Cas9 work better in almost all cells [73].…”

Section: Current Advantages and Limitationsmentioning

confidence: 99%

“…Thankfully, the latest research has revealed the Fanconi anemia pathway to make CRISPR/Cas9 work better in almost all cells [73]. For liver diseases requiring a high percentage of gene correction such as liver cancer and HBV, it is still unknown whether CRISPR can restore protein levels to a therapeutic threshold so increasing HDR efficiency is an essential task [25]. Great efforts have been made to address these impending issues, and the ongoing progress is encouraging, but much more is needed to fully realize the medical potential of CRISPR/Cas9.…”

Section: Current Advantages and Limitationsmentioning

confidence: 99%

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CRISPR/Cas9-related technologies in liver diseases: from feasibility to future diversity

Xu¹,

Li²,

Liu³

et al. 2020

Int. J. Biol. Sci.

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Liver diseases are one of the leading causes of mortality in the world, mainly caused by different etiological agents, alcohol consumption, viruses, drug intoxication, and malnutrition. The maturation of gene therapy has heralded new avenues for developing effective interventions for these diseases. Derived from a remarkable microbial defense system, clustered regularly interspaced short palindromic repeats/CRISPR-associated proteins 9 system (CRISPR/Cas9 system) is driving innovative applications from basic biology to biotechnology and medicine. Recently, the mutagenic function of CRISPR/Cas9 system has been widely adopted for genome and disease research. In this review, we describe the development and applications of CRISPR/Cas9 system on liver diseases for research or translational applications, while highlighting challenges as well as future avenues for innovation.

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CRISPR–Cas-related technologies in basic and translational liver research

Cited by 10 publications

References 10 publications

Long Noncoding RNA and Epithelial Mesenchymal Transition in Cancer

Long Noncoding RNA and Epithelial Mesenchymal Transition in Cancer

Adenine base editing in an adult mouse model of tyrosinaemia

CRISPR/Cas9-related technologies in liver diseases: from feasibility to future diversity

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