Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts (Q34556821)

3 August 2017

title

Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts (English)

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3 August 2017

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Waseem Qasim

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Adrian J Thrasher

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Wei-Li Di

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3 August 2017

Fernado Larcher

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3 August 2017

Ekaterina Semenova

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3 August 2017

Gill E Talbot

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3 August 2017

John I Harper

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3 August 2017

Marcela Del Rio

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3 August 2017

publication date

28 September 2010

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3 August 2017

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3 August 2017

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3 August 2017

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3 August 2017

page(s)

408-416

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describes a project that uses

3 August 2017

ImageJ

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11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC3034839/fullTextXML

based on heuristic

inferred from PubMed Central ID database lookup

cites work

Elastase 2 is expressed in human and mouse epidermis and impairs skin barrier function in Netherton syndrome through filaggrin and lipid misprocessing

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Corneodesmosomal cadherins are preferential targets of stratum corneum trypsin- and chymotrypsin-like hyperactivity in Netherton syndrome

7 July 2018

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LEKTI is localized in lamellar granules, separated from KLK5 and KLK7, and is secreted in the extracellular spaces of the superficial stratum granulosum

7 July 2018

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Spink5-deficient mice mimic Netherton syndrome through degradation of desmoglein 1 by epidermal protease hyperactivity

7 July 2018

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7 July 2018

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7 July 2018

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Human keratinocytes that express hTERT and also bypass a p16(INK4a)-enforced mechanism that limits life span become immortal yet retain normal growth and differentiation characteristics

7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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A heterozygous null mutation combined with the G1258A polymorphism of SPINK5 causes impaired LEKTI function and abnormal expression of skin barrier proteins

27 October 2018

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27 October 2018

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Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy

27 October 2018

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27 October 2018

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Durable and stratum-specific gene expression in epidermis

27 October 2018

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High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter

27 October 2018

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27 October 2018

Identifiers

DOI

10.1038/MT.2010.201

1 reference

3 August 2017

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3 August 2017

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