Targeted genetic repair: an emerging approach to genetic therapy (Q35190980)

7 August 2017

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title

Targeted genetic repair: an emerging approach to genetic therapy (English)

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7 August 2017

Bruce A Sullenger

1

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7 August 2017

language of work or name

English

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publication date

1 August 2003

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Journal of Clinical Investigation

7 August 2017

published in

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7 August 2017

volume

112

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7 August 2017

issue

3

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7 August 2017

page(s)

310-311

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Sequence-specific modification of genomic DNA by small DNA fragments

7 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Targeted gene repair -- in the arena

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

The potential for gene repair via triple helix formation

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Ribozyme-mediated revision of RNA and DNA

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Functional repair of a mutant chloride channel using a trans-splicing ribozyme

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

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

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Repairing the sickle cell mutation. I. Specific covalent binding of a photoreactive third strand to the mutated base pair

13 July 2018

1 reference

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Ribozyme-mediated trans-splicing of a trinucleotide repeat.

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

13 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Application of SFHR to gene therapy of monogenic disorders

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Expression of DeltaF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR.

27 September 2018

1 reference

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

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

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

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=166306

Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing

27 September 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Specific mutations induced by triplex-forming oligonucleotides in mice

1 reference

12 December 2020

Identifiers

DOI

10.1172/JCI200319419

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7 August 2017

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7 August 2017

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