Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models-Reference-Cited by-同舟云学术

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Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

Published:2018-09 Issue: Volume:12 Page:730-740
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Dulla Kalyan,Aguila Monica,Lane Amelia,Jovanovic Katarina,Parfitt David A.,Schulkens Iris,Chan Hee Lam,Schmidt Iris,Beumer Wouter,Vorthoren Lars,Collin Rob W.J.,Garanto Alejandro,Duijkers Lonneke,Brugulat-Panes Anna,Semo Ma’ayan,Vugler Anthony A.,Biasutto Patricia,Adamson Peter,Cheetham Michael E.

Funder

ProQR

Wellcome Trust

RP Fighting Blindness

Moorfields Eye Charity

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference50 articles.

1. Mutations in the CEP290 (NPHP6) gene are a frequent cause of Leber congenital amaurosis;den Hollander;Am. J. Hum. Genet.,2006

2. Basal exon skipping and genetic pleiotropy: a predictive model of disease pathogenesis;Drivas;Sci. Transl. Med.,2015

3. CEP290, a gene with many faces: mutation overview and presentation of CEP290base;Coppieters;Hum. Mutat.,2010

4. Leber congenital amaurosis associated with mutations in CEP290, clinical phenotype, and natural history in preparation for trials of novel therapies;Sheck;Ophthalmology,2018

5. Spectrum of NPHP6/CEP290 mutations in Leber congenital amaurosis and delineation of the associated phenotype;Perrault;Hum. Mutat.,2007

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