RNA repair: a novel approach to gene therapy (Q34079549)

30 July 2017

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title

RNA repair: a novel approach to gene therapy (English)

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30 July 2017

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Watanabe T

1

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PubMed ID

11072109

30 July 2017

Sullenger BA

series ordinal

2

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PubMed ID

11072109

30 July 2017

language of work or name

English

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publication date

1 November 2000

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Advanced Drug Delivery Reviews

30 July 2017

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30 July 2017

44

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30 July 2017

2-3

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30 July 2017

109-118

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Progress toward human gene therapy

30 July 2017

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Messenger RNA Splicing in Yeast: Clues to Why the Spliceosome Is a Ribonucleoprotein

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Biochemical Mechanisms of Constitutive and Regulated Pre-mRNA Splicing

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Gene therapy -- promises, problems and prospects

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Adenovirus-mediated gene transfer of the tumor suppressor, p53, induces apoptosis in postmitotic neurons

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Toward the therapeutic editing of mutated RNA sequences

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Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides

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Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs

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Two-step affinity purification of U7 small nuclear ribonucleoprotein particles using complementary biotinylated 2'-O-methyl oligoribonucleotides

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Self-splicing of group I introns

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Autocatalytic cyclization of an excised intervening sequence RNA is a cleavage–ligation reaction

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Fate of an intervening sequence ribonucleic acid: excision and cyclization of the Tetrahymena ribosomal ribonucleic acid intervening sequence in vivo

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One binding site determines sequence specificity of Tetrahymena pre-rRNA self-splicing, trans-splicing, and RNA enzyme activity

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Intermolecular exon ligation of the rRNA precursor of Tetrahymena: oligonucleotides can function as 5' exons

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Sequence-specific endoribonuclease activity of the Tetrahymena ribozyme: enhanced cleavage of certain oligonucleotide substrates that form mismatched ribozyme-substrate complexes

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Ribozyme-mediated repair of defective mRNA by targeted, trans-splicing

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Revising messages traveling along the cellular information superhighway

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Ribozyme-mediated repair of sickle beta-globin mRNAs in erythrocyte precursors

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Ribozyme-mediated trans-splicing of a trinucleotide repeat.

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Induction of wild-type p53 activity in human cancer cells by ribozymes that repair mutant p53 transcripts

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Evaluating and enhancing ribozyme reaction efficiency in mammalian cells

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Evaluating group I intron catalytic efficiency in mammalian cells

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Self-splicing of the Tetrahymena intron from mRNA in mammalian cells

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Implications of ribozyme kinetics for targeting the cleavage of specific RNA molecules in vivo: more isn't always better

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Catalysis of RNA cleavage by the Tetrahymena thermophila ribozyme. 2. Kinetic description of the reaction of an RNA substrate that forms a mismatch at the active site

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Optimizing the substrate specificity of a group I intron ribozyme

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Alteration of substrate specificity for the endoribonucleolytic cleavage of RNA by the Tetrahymena ribozyme

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Trans -splicing ribozymes for targeted gene delivery 1 1Edited by J. Karn

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Identifiers

DOI

1 reference

30 July 2017

1 reference

PubMed ID

11072109