Rescue of common exon‐skipping mutations in cystic fibrosis with modified U1 snRNAs-Reference-Cited by-同舟云学术

Rescue of common exon‐skipping mutations in cystic fibrosis with modified U1 snRNAs

Published:2020-10-14 Issue:12 Volume:41 Page:2143-2154
ISSN:1059-7794
Container-title:Human Mutation
language:en
Short-container-title:Human Mutation

Author:

Donegà Stefano¹^ORCID,Rogalska Malgorzata Ewa¹^ORCID,Pianigiani Giulia¹^ORCID,Igreja Susana²^ORCID,Amaral Margarida Duarte²^ORCID,Pagani Franco¹^ORCID

Affiliation:

1. Human Molecular Genetics ICGEB – International Center for Genetic Engineering and Biotechnology Trieste Italy

2. BioISI – Biosystems and Integrative Sciences Institute, Faculty of Sciences University of Lisbon Lisbon Portugal

Funder

Fondazione per la Ricerca sulla Fibrosi Cistica

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/humu.24116

Reference76 articles.

1. RNA Structure Modulates Splicing Efficiency at the Human Immunodeficiency Virus Type 1 Major Splice Donor

2. Combined Computational-Experimental Analyses ofCFTRExon Strength Uncover Predictability of Exon-Skipping Level

3. An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects

4. TDP43 depletion rescues aberrant CFTR exon 9 skipping

5. An engineered U 1 small nuclear RNA rescues splicing‐defective coagulation F 7 gene expression in mice

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2. Development of Engineered-U1 snRNA Therapies: Current Status;International Journal of Molecular Sciences;2023-09-27

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