Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation-Reference-Cited by-同舟云学术

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Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation

Published:2019-12 Issue: Volume:18 Page:123-130
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Breuel Saskia,Vorm Mariann,Bräuer Anja U.,Owczarek-Lipska Marta,Neidhardt John

Funder

Faculty of Medicine and Health Science at the University of Oldenburg, Germany

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference46 articles.

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2. Clinical and genetic epidemiology of Bardet-Biedl syndrome in Newfoundland: a 22-year prospective, population-based, cohort study;Moore;Am. J. Med. Genet. A.,2005

3. A novel founder BBS1 mutation explains a unique high prevalence of Bardet-Biedl syndrome in the Faroe Islands;Hjortshøj;Br. J. Ophthalmol.,2009

4. Bardet-Biedl Syndrome;Suspitsin;Mol. Syndromol.,2016

5. Managing Bardet-Biedl Syndrome-Now and in the Future;Forsythe;Front Pediatr.,2018

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1. Characterization and Engineered U1 snRNA Rescue of Splicing Variants in a Turkish Neurodevelopmental Disease Cohort;Human Mutation;2024-05-28

2. Splicing Modulation via Antisense Oligonucleotides in Recessive Dystrophic Epidermolysis Bullosa;International Journal of Molecular Sciences;2024-01-07

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

4. In Vivo Efficacy and Safety Evaluations of Therapeutic Splicing Correction Using U1 snRNA in the Mouse Retina;Cells;2023-03-21

5. Counteracting the Common Shwachman–Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing;International Journal of Molecular Sciences;2023-02-16

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