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Comprehensive Assessment of ICFTR/I Modulators’ Therapeutic Efficiency for N1303K Variant

Comprehensive Assessment of ICFTR/I Modulators’ Therapeutic Efficiency for N1303K Variant

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_gale_infotracmisc_A786437029

Comprehensive Assessment of ICFTR/I Modulators’ Therapeutic Efficiency for N1303K Variant

About this item

Full title

Comprehensive Assessment of ICFTR/I Modulators’ Therapeutic Efficiency for N1303K Variant

Publisher

MDPI AG

Journal title

International journal of molecular sciences, 2024-02, Vol.25 (5)

Language

English

Formats

Publication information

Publisher

MDPI AG

More information

Scope and Contents

Contents

p.Asn1303Lys (N1303K) is a common missense variant of the CFTR gene, causing cystic fibrosis (CF). In this study, we initially evaluated the influence of CFTR modulators on the restoration of N1303K-CFTR function using intestinal organoids derived from four CF patients expressing the N1303K variant. The forskolin-induced swelling assay in organoids...

Alternative Titles

Full title

Comprehensive Assessment of ICFTR/I Modulators’ Therapeutic Efficiency for N1303K Variant

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_gale_infotracmisc_A786437029

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_gale_infotracmisc_A786437029

Other Identifiers

ISSN

1422-0067

DOI

10.3390/ijms25052770

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