Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides-Reference-Cited by-同舟云学术

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Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides

Published:2015-12-02 Issue:2 Volume:37 Page:209-215
ISSN:1059-7794
Container-title:Human Mutation
language:en
Short-container-title:Human Mutation

Author:

Igreja Susana¹,Clarke Luka A.¹,Botelho Hugo M.¹,Marques Luís¹,Amaral Margarida D.¹

Affiliation:

1. University of Lisboa; Faculty of Sciences; BioISI - Biosystems & Integrative Sciences Institute; Lisboa Portugal

Funder

Foundation of Science and Technology, Portugal (FCT/MCTES/PIDDAC)

FCT/MCTES/PIDDAC, Portugal

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/humu.22931/fullpdf

Reference31 articles.

1. Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients;Amaral;J Intern Med,2015

2. EGCG corrects aberrant splicing of IKAP mRNA in cells from patients with familial dysautonomia;Anderson;Biochem Biophys Res Commun,2003

3. New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls;Bell;Pharmacol Ther,2015

4. Protein traffic disorders: an effective high-throughput fluorescence microscopy pipeline for drug discovery;Botelho;Sci Rep,2015

5. Aberrant CFTR-dependent HCO3-transport in mutations associated with cystic fibrosis;Choi;Nature,2001

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4. Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C;Molecular Therapy - Nucleic Acids;2023-03

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