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Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation...

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation...

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2505394841

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus

About this item

Full title

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus

Publisher

Cold Spring Harbor: Cold Spring Harbor Laboratory Press

Journal title

bioRxiv, 2021-02

Language

English

Formats

Publication information

Publisher

Cold Spring Harbor: Cold Spring Harbor Laboratory Press

More information

Scope and Contents

Contents

Abstract Cystic fibrosis (CF) is a monogenic disease caused by impaired production and/or function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Although we have previously shown correction of the most common pathogenic mutation, there are many other pathogenic mutations throughout the CF gene. An autologous airway stem...

Alternative Titles

Full title

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_proquest_journals_2505394841

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2505394841

Other Identifiers

ISSN

2692-8205

E-ISSN

2692-8205

DOI

10.1101/2021.02.26.432961

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