当前位置： SCI文献检索 > Molecular Therapy-Nucleic Acids期刊下所有文献 > CRISPR-Cas9 System as a Versatile Tool for Genome Engineering in Human Cells.

CRISPR-Cas9 System as a Versatile Tool for Genome Engineering in Human Cells.

Abstract：

:Targeted nucleases are influential instruments for intervening in genome revision with great accuracy. RNA-guided Cas9 nucleases produced from clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems have noticeably altered the means to modify the genomes of distinct organisms. They can be notably used to facilitate effective genome manipulation in eukaryotic cells by clearly detailing a 20-nt targeting sequence inside its directed RNA. We discuss the most recent advancements in the molecular basis of the type II CRISPR/Cas system and encapsulate applications and elements affecting its use in human cells. We also propose possible applications covering its uses ranging from basic science to implementation in the clinic.

journal_name

Mol Ther Nucleic Acids

journal_title

Molecular therapy. Nucleic acids

authors

Wang X,Huang X,Fang X,Zhang Y,Wang W

doi

10.1038/mtna.2016.95

subject

Has Abstract

pub_date

2016-11-15 00:00:00

pages

e388

issue

11

issn

2162-2531

pii

mtna201695

journal_volume

5

pub_type

杂志文章,评审

相关文献

Molecular Therapy-Nucleic Acids文献大全
  • Development of a Facile Approach for Generating Chemically Modified CRISPR/Cas9 RNA.

    abstract：:The RNA-guided, modified type II prokaryotic CRISPR with CRISPR-associated proteins (CRISPR/Cas9) system represents a simple gene-editing platform with applications in biotechnology and also potentially as a therapeutic modality. The system requires a small guide RNA (sgRNA) and a catalytic Cas9 protein to induce non-...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2020.01.004

    authors： Scott T,Soemardy C,Morris KV

    更新日期：2020-03-06 00:00:00

  • COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites.

    abstract：:Precise genome editing using engineered nucleases can significantly facilitate biological studies and disease treatment. In particular, clustered regularly interspaced short palindromic repeats (CRISPR) with CRISPR-associated (Cas) proteins are a potentially powerful tool for modifying a genome by targeted cleavage of...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2014.64

    authors： Cradick TJ,Qiu P,Lee CM,Fine EJ,Bao G

    更新日期：2014-12-02 00:00:00

  • Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification.

    abstract：:The acute hepatic porphyrias are caused by inherited enzymatic deficiencies in the heme biosynthesis pathway. Induction of the first enzyme 5-aminolevulinic acid synthase 1 (ALAS1) by triggers such as fasting or drug exposure can lead to accumulation of neurotoxic heme intermediates that cause disease symptoms. We hav...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2015.36

    authors： Chan A,Liebow A,Yasuda M,Gan L,Racie T,Maier M,Kuchimanchi S,Foster D,Milstein S,Charisse K,Sehgal A,Manoharan M,Meyers R,Fitzgerald K,Simon A,Desnick RJ,Querbes W

    更新日期：2015-11-03 00:00:00

  • miR-134: A Human Cancer Suppressor?

    abstract：:MicroRNAs (miRNAs) are small noncoding RNAs approximately 20-25 nt in length, which play crucial roles through directly binding to corresponding 3' UTR of targeted mRNAs. It has been reported that miRNAs are involved in numerous of diseases, including cancers. Recently, miR-134 has been identified to dysregulate in ha...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章,评审

    doi：10.1016/j.omtn.2016.11.003

    authors： Pan JY,Zhang F,Sun CC,Li SJ,Li G,Gong FY,Bo T,He J,Hua RX,Hu WD,Yuan ZP,Wang X,He QQ,Li DJ

    更新日期：2017-03-17 00:00:00

  • Downregulation of MicroRNA-222 Reduces Insulin Resistance in Rats with PCOS by Inhibiting Activation of the MAPK/ERK Pathway via Pten.

    abstract：:Polycystic ovary syndrome (PCOS), characterized by the dysfunction of endocrine metabolism, is a common disease among women. Insulin (INS) resistance (IR) is considered as an obstruction to effective PCOS treatment. Here, we aimed to explore the mechanism by which microRNA-222 (miR-222) affects IR in PCOS via Pten. Qu...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2020.07.014

    authors： Ye H,Liu XJ,Hui Y,Liang YH,Li CH,Wan Q

    更新日期：2020-07-10 00:00:00

  • Osteopontin Upregulates Col IV Expression by Repressing miR-29a in Human Retinal Capillary Endothelial Cells.

    abstract：:Abnormal synthesis of extracellular matrix (ECM), especially collagen type IV (Col IV), in human retinal capillary endothelial cells (HRCECs) and resultant basement membrane (BM) thickening is the most prominent and characteristic feature of early diabetic retinopathy (DR). Osteopontin (OPN) has been shown to play an ...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2020.02.001

    authors： Duan P,Chen S,Zeng Y,Xu H,Liu Y

    更新日期：2020-06-05 00:00:00

  • circINSR Promotes Proliferation and Reduces Apoptosis of Embryonic Myoblasts by Sponging miR-34a.

    abstract：:As a diverse and abundant class of endogenous RNAs, circular RNAs (circRNAs) participate in processes including cell proliferation and apoptosis. Nevertheless, few researchers have investigated the function of circRNAs in bovine muscle development. Based on existing sequencing data, we identified circINSR. The localiz...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.12.032

    authors： Shen X,Zhang X,Ru W,Huang Y,Lan X,Lei C,Chen H

    更新日期：2020-03-06 00:00:00

  • Long Noncoding RNA ASLNC07322 Functions in VEGF-C Expression Regulated by Smad4 during Colon Cancer Metastasis.

    abstract：:Deletion and mutation of the Smad4 gene are favorable events for the progression of colon cancer, which is related to the negative regulation of vascular endothelial growth factor C (VEGF-C). However, the regulatory mechanism between Smad4 and VEGF-C remains unclear. We reported first that Smad4 can increase the trans...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.10.012

    authors： Li X,Lv X,Li Z,Li C,Li X,Xiao J,Liu B,Yang H,Zhang Y

    更新日期：2019-12-06 00:00:00

  • An innovative data analysis strategy for accurate next-generation sequencing detection of tumor mitochondrial DNA mutations.

    abstract：:Next-generation sequencing technology has been commonly applied to detect mitochondrial DNA (mtDNA) mutations, which are reported to be strongly associated with cancers. However, several key challenges still exist regarding bioinformatics analysis of mtDNA sequencing data that greatly affect the detection accuracy of ...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2020.11.002

    authors： Guo S,Zhou K,Yuan Q,Su L,Liu Y,Ji X,Gu X,Guo X,Xing J

    更新日期：2020-11-11 00:00:00

  • In Vivo Selection Against Human Colorectal Cancer Xenografts Identifies an Aptamer That Targets RNA Helicase Protein DHX9.

    abstract：:The ability to selectively target disease-related tissues with molecules is critical to the design of effective therapeutic and diagnostic reagents. Recognizing the differences between the in vivo environment and in vitro conditions, we employed an in vivo selection strategy to identify RNA aptamers (targeting motifs)...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2016.27

    authors： Mi J,Ray P,Liu J,Kuan CT,Xu J,Hsu D,Sullenger BA,White RR,Clary BM

    更新日期：2016-04-26 00:00:00

  • Progress in RNAi-mediated Molecular Therapy of Acute and Chronic Myeloid Leukemia.

    abstract：:Leukemias arise from genetic alterations in normal hematopoietic stem or progenitor cells, leading to impaired regulation of proliferation, differentiation, apoptosis, and survival of the transformed cells. With the advent of RNA interference (RNAi) and the short interfering RNA (siRNA) as its pharmacological mediator...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章,评审

    doi：10.1038/mtna.2015.13

    authors： Landry B,Valencia-Serna J,Gul-Uludag H,Jiang X,Janowska-Wieczorek A,Brandwein J,Uludag H

    更新日期：2015-05-12 00:00:00

  • Functional Significance and Therapeutic Potential of miR-15a Mimic in Pancreatic Ductal Adenocarcinoma.

    abstract：:Treatment of pancreatic ductal adenocarcinoma (PDAC) remains a clinical challenge. There is an urgent need to develop novel strategies to enhance survival and improve patient prognosis. MicroRNAs (miRNAs) play critical roles as oncogenes or tumor suppressors in the regulation of cancer development and progression. In ...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.11.010

    authors： Guo S,Fesler A,Huang W,Wang Y,Yang J,Wang X,Zheng Y,Hwang GR,Wang H,Ju J

    更新日期：2020-03-06 00:00:00

  • Circulating lncRNA UCA1 Promotes Malignancy of Colorectal Cancer via the miR-143/MYO6 Axis.

    abstract：:Exosomes mediate cell-cell crosstalk in cancer progression by transferring a variety of biomolecules, including long noncoding RNAs (lncRNAs). Long non-coding RNA urothelial carcinoma-associated (UCA1) is a well-known lncRNA associated with the development and progression of various cancers, including colorectal cance...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.12.009

    authors： Luan Y,Li X,Luan Y,Zhao R,Li Y,Liu L,Hao Y,Oleg Vladimir B,Jia L

    更新日期：2020-03-06 00:00:00

  • E-selectin Targeting PEGylated-thioaptamer Prevents Breast Cancer Metastases.

    abstract：:E-selectin is an adhesion molecule expressed on the luminal surface of inflamed blood vessels that mediates hematogenous metastasis by assisting shear-resistant adhesion of circulating tumor cells to the vessel surface under dynamic blood flow. Previously, we developed an E-selectin antagonistic thioaptamer (ESTA) for...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2016.103

    authors： Morita Y,Kamal M,Kang SA,Zhang R,Lokesh GL,Thiviyanathan V,Hasan N,Woo S,Zhao D,Leslie M,Suh S,Razaq W,Rui H,Gorenstein DG,Volk DE,Tanaka T

    更新日期：2016-12-13 00:00:00

  • Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting.

    abstract：:Engineered nuclease-mediated gene targeting through homologous recombination (HR) in hematopoietic stem and progenitor cells (HSPCs) has the potential to treat a variety of genetic hematologic and immunologic disorders. Here, we identify critical parameters to reproducibly achieve high frequencies of RNA-guided (singl...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2018.04.017

    authors： Charlesworth CT,Camarena J,Cromer MK,Vaidyanathan S,Bak RO,Carte JM,Potter J,Dever DP,Porteus MH

    更新日期：2018-09-07 00:00:00

  • Studying Interactions between 2'-O-Me-Modified Inhibitors and MicroRNAs Utilizing Microscale Thermophoresis.

    abstract：:Besides the acquisition of pharmacokinetic parameters of antisense oligonucleotide microRNA (miRNA) inhibitors, such as measuring in vivo concentration, their pharmacodynamic characteristics are also of interest. An emerging and straightforward method for studying molecular interactions is microscale thermophoresis (M...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.08.019

    authors： Herkt M,Batkai S,Thum T

    更新日期：2019-12-06 00:00:00

  • MicroRNA let-7i Inhibits Histone Lysine Demethylase KDM5B to Halt Esophageal Cancer Progression.

    abstract：:Recent studies have suggested that microRNA let-7i is a tumor suppressor in human cancers, including esophageal cancer, but its underlying mechanism is not yet fully understood. We investigated the role and mechanisms of let-7i in the progression of esophageal cancer. We first showed that let-7i was downregulated in e...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2020.09.012

    authors： Yang Y,Li W,Wei B,Wu K,Liu D,Zhu D,Zhang C,Wen F,Fan Y,Zhao S

    更新日期：2020-09-16 00:00:00

  • The Antibiotic-free pFAR4 Vector Paired with the Sleeping Beauty Transposon System Mediates Efficient Transgene Delivery in Human Cells.

    abstract：:The anti-angiogenic and neurogenic pigment epithelium-derived factor (PEDF) demonstrated a potency to control choroidal neovascularization in age-related macular degeneration (AMD) patients. The goal of the present study was the development of an efficient and safe technique to integrate, ex vivo, the PEDF gene into r...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2017.12.017

    authors： Pastor M,Johnen S,Harmening N,Quiviger M,Pailloux J,Kropp M,Walter P,Ivics Z,Izsvák Z,Thumann G,Scherman D,Marie C

    更新日期：2018-06-01 00:00:00

  • Identification and Characterization of an eIF4e DNA Aptamer That Inhibits Proliferation With High Throughput Sequencing.

    abstract：:Development of DNA aptamer screens that are both simple and informative can increase the success rate of DNA aptamer selection and induce greater adoption. High eIF4e levels contribute to malignancies, thus eIF4e presents itself as a valuable target for DNA aptamer-based inhibition screen. Here, we demonstrate a metho...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2014.70

    authors： Guo WM,Kong KW,Brown CJ,Quah ST,Yeo HL,Hoon S,Seow Y

    更新日期：2014-12-16 00:00:00

  • Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS.

    abstract：:Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the CNS. This popularity originates from a high safety record and the longevity of transgene expression in neurons. Nevertheless, clinical efficacy for CNS indications is lacking, and one reason for this is the relatively limited sprea...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2017.06.011

    authors： Kanaan NM,Sellnow RC,Boye SL,Coberly B,Bennett A,Agbandje-McKenna M,Sortwell CE,Hauswirth WW,Boye SE,Manfredsson FP

    更新日期：2017-09-15 00:00:00

  • MSTN Attenuates Cardiac Hypertrophy through Inhibition of Excessive Cardiac Autophagy by Blocking AMPK /mTOR and miR-128/PPARγ/NF-κB.

    abstract：:Cardiac hypertrophy, a response of the heart to increased workload, is a major risk factor for heart failure. Myostatin (MSTN) is an inhibitor of myogenesis, regulating the number and size of skeletal myocytes. In recent years, cardiomyocyte autophagy also has been considered to be involved in controlling the hypertro...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.12.003

    authors： Qi H,Ren J,Ba L,Song C,Zhang Q,Cao Y,Shi P,Fu B,Liu Y,Sun H

    更新日期：2020-03-06 00:00:00

  • Delivery of Quantum Dot-siRNA Nanoplexes in SK-N-SH Cells for BACE1 Gene Silencing and Intracellular Imaging.

    abstract：:The fluorescent quantum dots (QDs) delivered small interfering RNAs (siRNAs) targeting β-secretase (BACE1) to achieve high transfection efficiency of siRNAs and reduction of β-amyloid (Aβ) in nerve cells. The CdSe/ZnS QDs with the conjugation of amino-polyethylene glycol (PEG) were synthesized. Negatively charged siRN...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2012.11

    authors： Li S,Liu Z,Ji F,Xiao Z,Wang M,Peng Y,Zhang Y,Liu L,Liang Z,Li F

    更新日期：2012-04-24 00:00:00

  • Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.

    abstract：:The knockdown of myostatin, a negative regulator of skeletal muscle mass may have important implications in disease conditions accompanied by muscle mass loss like cancer, HIV/AIDS, sarcopenia, muscle atrophy, and Duchenne muscular dystrophy (DMD). In DMD patients, where major muscle loss has occurred due to a lack of...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2012.54

    authors： Malerba A,Kang JK,McClorey G,Saleh AF,Popplewell L,Gait MJ,Wood MJ,Dickson G

    更新日期：2012-12-18 00:00:00

  • A Kinetic Model Explains Why Shorter and Less Affine Enzyme-recruiting Oligonucleotides Can Be More Potent.

    abstract：:Antisense oligonucleotides complementary to RNA targets promise generality and ease of drug design. The first systemically administered antisense drug was recently approved for treatment and others are in clinical development. Chemical modifications that increase the hybridization affinity of oligonucleotides are reas...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2013.72

    authors： Pedersen L,Hagedorn PH,Lindholm MW,Lindow M

    更新日期：2014-02-18 00:00:00

  • A Small RNA Transforms the Multidrug Resistance of Pseudomonas aeruginosa to Drug Susceptibility.

    abstract：:Bacteria with multiple drug resistance (MDR) have become a global issue worldwide, and hundreds of thousands of people's lives are threatened every year. The emergence of novel MDR strains and insufficient development of new antimicrobial agents are the major reasons that limit the choice of antibiotics for the treatm...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.02.011

    authors： Law COK,Huang C,Pan Q,Lee J,Hao Q,Chan TF,Lo NWS,Ang IL,Koon A,Ip M,Chan E,Lau TCK

    更新日期：2019-06-07 00:00:00

  • Preclinical in vivo evaluation of the safety of a multi-shRNA-based gene therapy against HIV-1.

    abstract：:Highly active antiretroviral therapy (HAART) has significantly improved the quality of life and the life expectancy of HIV-infected individuals. Still, drug-induced side effects and emergence of drug-resistant viral variants remain important issues that justify the exploration of alternative therapeutic options. One s...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2013.48

    authors： Centlivre M,Legrand N,Klamer S,Liu YP,Jasmijn von Eije K,Bohne M,Rijnstra ES,Weijer K,Blom B,Voermans C,Spits H,Berkhout B

    更新日期：2013-09-03 00:00:00

  • Lipidoid Nanoparticles Containing PD-L1 siRNA Delivered In Vivo Enter Kupffer Cells and Enhance NK and CD8(+) T Cell-mediated Hepatic Antiviral Immunity.

    abstract：:Effective clinical application of antiviral immunotherapies necessitates enhancing the functional state of natural killer (NK) and CD8(+) T cells. An important mechanism for the establishment of viral persistence in the liver is the activation of the PD-1/PD-L1 inhibitory pathway. To examine the role of hepatic myeloi...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2012.63

    authors： Dolina JS,Sung SS,Novobrantseva TI,Nguyen TM,Hahn YS

    更新日期：2013-02-19 00:00:00

  • Cross-Species Suppression of Hepatoma Cell Growth and Migration by a Schistosoma japonicum MicroRNA.

    abstract：:Schistosoma japonicum eggs trapped in host liver secretes microRNA (miRNA)-containing extracellular vesicles (EVs) that can be transferred to host cells. Recent studies demonstrated that miRNAs derived from plants can modulate gene expression and phenotype of mammalian cells in a cross-kingdom manner. In this study, w...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2019.09.006

    authors： Lin Y,Zhu S,Hu C,Wang J,Jiang P,Zhu L,Li Z,Wang S,Zhang Y,Xu X,Pan W

    更新日期：2019-12-06 00:00:00

  • RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect.

    abstract：:Non-coding uridine-rich small nuclear RNAs (UsnRNAs) have emerged in recent years as effective tools for exon skipping for the treatment of Duchenne muscular dystrophy (DMD), a degenerative muscular genetic disorder. We recently showed the high capacity of a recombinant adeno-associated virus (rAAV)-U7snRNA vector to ...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1016/j.omtn.2017.12.008

    authors： Domenger C,Allais M,François V,Léger A,Lecomte E,Montus M,Servais L,Voit T,Moullier P,Audic Y,Le Guiner C

    更新日期：2018-03-02 00:00:00

  • Neutrophils are Essential in Short Hairpin RNA of Indoleamine 2,3- Dioxygenase Mediated-antitumor Efficiency.

    abstract：:Indoleamine 2,3-dioxygenase (IDO) is a rate limiting enzyme in tryptophan-degrading pathways and IDO activity results in immune suppression. Targeting IDO is a strategy of cancer immunotherapies. Our previous studies demonstrate that delivery of short hairpin against IDO (IDO shRNA) suppresses tumor growth and increas...

    journal_title：Molecular therapy. Nucleic acids

    pub_type： 杂志文章

    doi：10.1038/mtna.2016.105

    authors： Liu KT,Liu YH,Liu HL,Chong IW,Yen MC,Kuo PL

    更新日期：2016-12-06 00:00:00