(Q40141498)

17 September 2017

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery (English)

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Sleeping Beauty transposon system

17 September 2017

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10

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17 September 2017

Hatem Zayed

1

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David L. Wiest

object named as

David L Wiest

8

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Bruce R Blazar

11

object named as

Bruce R Blazar

1 reference

17 September 2017

9

R Scott McIvor

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17 September 2017

Lily Xia

2

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17 September 2017

Anton Yerich

3

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17 September 2017

Stephen R Yant

4

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17 September 2017

Mark A Kay

5

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17 September 2017

M Puttaraju

6

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17 September 2017

Gerard J McGarrity

7

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17 September 2017

24 April 2007

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17 September 2017

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17 September 2017

15

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17 September 2017

7

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17 September 2017

1273-1279

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

17 September 2017

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Sleeping beauty transposon-mediated gene therapy for prolonged expression

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Awakening gene therapy with Sleeping Beauty transposons

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Sleeping Beauty, a wide host-range transposon vector for genetic transformation in vertebrates

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Gene transfer into genomes of human cells by the sleeping beauty transposon system.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Cytokines and immunodeficiency diseases: critical roles of the gamma(c)-dependent cytokines interleukins 2, 4, 7, 9, 15, and 21, and their signaling pathways

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Nonsense mutation at Tyr-4046 in the DNA-dependent protein kinase catalytic subunit of severe combined immune deficiency mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Regulation and mechanisms of mammalian double-strand break repair

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

The scid mutation in mice causes a general defect in DNA repair

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Pluripotency of mesenchymal stem cells derived from adult marrow

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Real-time in vivo imaging of stem cells following transgenesis by transposition.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Pyrosequencing: history, biochemistry and future

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

RNA repair using spliceosome-mediated RNA trans-splicing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Sleeping beauty transposition: biology and applications for molecular therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Nonhomologous-End-Joining Factors Regulate DNA Repair Fidelity during Sleeping Beauty Element Transposition in Mammalian Cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Healing the wounds inflicted by sleeping beauty transposition by double-strand break repair in mammalian somatic cells.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Promiscuity of pre-mRNA spliceosome-mediated trans splicing: a problem for gene therapy?

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Homozygosity for a missense mutation (G20R) associated with neonatal onset adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID).

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of JAK3-deficient mice without myeloablation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Hematopoietic reconstitution by multipotent adult progenitor cells: precursors to long-term hematopoietic stem cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

RETRACTED: Purification and ex vivo expansion of postnatal human marrow mesodermal progenitor cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Murine cell line SX9 bearing a mutation in the dna-pkcs gene exhibits aberrant V(D)J recombination not only in the coding joint but also in the signal joint

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Tec kinases mediate sustained calcium influx via site-specific tyrosine phosphorylation of the phospholipase Cgamma Src homology 2-Src homology 3 linker

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Splinkerettes--improved vectorettes for greater efficiency in PCR walking.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300178

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300178

1 reference

17 September 2017

1 reference