Search Results - "Cheng, Congsheng"
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High-fidelity SaCas9 identified by directional screening in human cells
Published in PLoS biology (09-07-2020)“…CRISPR-Staphylococcus aureus Cas9 (CRISPR-SaCas9) has been harnessed as an effective in vivo genome-editing tool to manipulate genomes. However, off-target…”
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In vivo SELEX for Identification of Brain-penetrating Aptamers
Published in Molecular therapy. Nucleic acids (01-01-2013)“…The physiological barriers of the brain impair drug delivery for treatment of many neurological disorders. One delivery approach that has not been investigated…”
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Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching
Published in Nature communications (06-05-2023)“…Alternative splicing of neuronal genes is controlled partly by the coordinated action of polypyrimidine tract binding proteins (PTBPs). While PTBP1 is…”
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Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing
Published in HGG advances (12-01-2023)“…Huntington’s disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG trinucleotide repeat expansions in exon-1 of huntingtin (HTT)…”
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Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing
Published in HGG advances (13-07-2023)Get full text
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Potent inhibition of human influenza H5N1 virus by oligonucleotides derived by SELEX
Published in Biochemical and biophysical research communications (15-02-2008)“…New therapeutics are urgently needed for the treatment of pandemic influenza caused by H5N1 influenza virus mutants. Aptamer was a promising candidate for…”
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Inhibitory effect of small interfering RNA specific for a novel candidate target in PB1 gene of influenza A virus
Published in Journal of drug targeting (01-02-2009)“…Influenza, mainly caused by influenza virus, is becoming one of the major concerns in the world. Limitation in vaccines necessitates the urgent development of…”
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The long non-coding RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease gene-dependent toxicities
Published in Human molecular genetics (15-12-2018)“…Abstract Polyglutamine (polyQ) repeat diseases are a class of neurodegenerative disorders caused by CAG-repeat expansion. There are diverse cellular mechanisms…”
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Elucidation of transcriptome-wide microRNA binding sites in human cardiac tissues by Ago2 HITS-CLIP
Published in Nucleic acids research (06-09-2016)“…MicroRNAs (miRs) have emerged as key biological effectors in human health and disease. These small noncoding RNAs are incorporated into Argonaute (Ago)…”
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Efficient Human Genome Editing Using SaCas9 Ribonucleoprotein Complexes
Published in Biotechnology journal (01-07-2019)“…Genome editing using RNA‐guided nucleases in their ribonucleoprotein (RNP) form represents a promising strategy for gene modification and therapy because they…”
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Erratum: Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing
Published in HGG advances (13-07-2023)“…[This corrects the article DOI: 10.1016/j.xhgg.2022.100146.]…”
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Abstract 17984: No Longer Silent: A Synonymous Coding SNP Modifying a Novel MicroRNA-24: SCN5A Interaction Associates With Non-Arrhythmic Death in Heart Failure
Published in Circulation (New York, N.Y.) (10-11-2015)“…Mutations that disrupt the coding sequence of Nav1.5, the cardiac Na+ channel encoded by SCN5A, cause inherited arrhythmias and cardiomyopathy. Changes in…”
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Construction and delivery of gene therapy vector containing soluble TNFα receptor-IgGFc fusion gene for the treatment of allergic rhinitis
Published in Cytokine (Philadelphia, Pa.) (01-12-2006)“…Tumor necrosis factor α plays primary role in the pathogenesis of inflammatory diseases. TNFα is essential for antigen-specific IgE production and for the…”
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Construction and delivery of gene therapy vector containing soluble TNFalpha receptor-IgGFc fusion gene for the treatment of allergic rhinitis
Published in Cytokine (Philadelphia, Pa.) (01-12-2006)“…Tumor necrosis factor alpha plays primary role in the pathogenesis of inflammatory diseases. TNFalpha is essential for antigen-specific IgE production and for…”
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