(Q34807121)

4 August 2017

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title

Oligonucleotide-mediated gene therapy for muscular dystrophies (English)

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4 August 2017

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1

Thomas A Rando

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4 August 2017

publication date

1 October 2002

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4 August 2017

published in

Neuromuscular Disorders

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4 August 2017

volume

12 Suppl 1

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4 August 2017

page(s)

S55-60

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The potential of nucleic acid repair in functional genomics

4 August 2017

cites work

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Gene correction by RNA-DNA oligonucleotides

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Gene repair using chimeric RNA/DNA oligonucleotides

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Gene targeting via triple-helix formation

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Targeted correction of a defective selectable marker gene in human epithelial cells by small DNA fragments

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Targeted gene correction by small single-stranded oligonucleotides in mammalian cells

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Spliceosome-mediated RNA trans-splicing as a tool for gene therapy

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Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides

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Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides

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In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide

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Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides

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7 January 2021

Dystrophin gene transcripts skipping the mdx mutation

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Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells

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Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.

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In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement

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Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

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CD45 fraction bone marrow cells as potential delivery vehicles for genetically corrected dystrophin loci.

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A role for RNA synthesis in homologous pairing events

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Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide

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Targeted manipulation of maize genes in vivo using chimeric RNA/DNA oligonucleotides

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A tool for functional plant genomics: chimeric RNA/DNA oligonucleotides cause in vivo gene-specific mutations

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Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide

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7 January 2021

Targeted gene conversion in a mammalian CD34+-enriched cell population using a chimeric RNA/DNA oligonucleotide

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7 January 2021

In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides

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7 January 2021

Stable and inheritable changes in genotype and phenotype of albino melanocytes induced by an RNA-DNA oligonucleotide

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7 January 2021

Correction of the UDP-glucuronosyltransferase gene defect in the gunn rat model of crigler-najjar syndrome type I with a chimeric oligonucleotide

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7 January 2021

Localized in vivo genotypic and phenotypic correction of the albino mutation in skin by RNA-DNA oligonucleotide

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7 January 2021

Different frequency of gene targeting events by the RNA-DNA oligonucleotide among epithelial cells

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Nucleotide exchange in genomic DNA of rat hepatocytes using RNA/DNA oligonucleotides. Targeted delivery of liposomes and polyethyleneimine to the asialoglycoprotein receptor

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Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA.DNA oligonucleotide

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7 January 2021

The site-specific correction of genetic defects

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7 January 2021

Persistent failures in gene repair

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7 January 2021

Failure to achieve gene conversion with chimeric circular oligonucleotides: potentially misleading PCR artifacts observed

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7 January 2021

A plausible mechanism for gene correction by chimeric oligonucleotides

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7 January 2021

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7 January 2021

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7 January 2021

Gene correction of the apolipoprotein (Apo) E2 phenotype to wild-type ApoE3 by in situ chimeraplasty

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

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7 January 2021

In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides

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7 January 2021

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7 January 2021

Selection and use of ligands for receptor-mediated gene delivery to myogenic cells

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7 January 2021

Cell penetrating PNA constructs regulate galanin receptor levels and modify pain transmission in vivo

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7 January 2021

Inhibition of telomerase activity by a cell-penetrating peptide nucleic acid construct in human melanoma cells

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7 January 2021

In vivo protein transduction: delivery of a biologically active protein into the mouse

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7 January 2021

A sequence-specific gene correction by an RNA-DNA oligonucleotide in mammalian cells characterized by transfection and nuclear extract using a lacZ shuttle system

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7 January 2021

10.1016/S0960-8966(02)00083-4

Identifiers

DOI

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4 August 2017

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