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Open access

Research article

First published 2016

Small Molecules

Saida Ortolano

Journal of Inborn Errors of Metabolism and Screening

Retricted access

Other

First published 2010-9

Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/ Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)

Richard S. Finkel

Journal of Child Neurology

Open access

Research article

First published 2019-08

Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations

Lulu Huang, Mariam Aghajan, Tianna Quesenberry [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2006-05

Chimeric Adeno-Associated Virus/Antisense U1 Small Nuclear RNA Effectively Rescues Dystrophin Synthesis and Muscle Function by Local Treatment of mdx Mice

Michela Alessandra Denti, Alessandro Rosa, Giuseppe D'Antona [...]

Human Gene Therapy

Open access

Research article

First published 2013

A Statistical Method without Training Step for the Classification of Coding Frame in Transcriptome Sequences

Nicolas Carels, Diego Frías

Bioinformatics and Biology Insights

Open access

Research article

First published 2020-7

Targeting translational read-through of premature termination mutations in BMPR2 with PTC124 for pulmonary arterial hypertension

Lu Long, Xudong Yang, Mark Southwood [...]

Pulmonary Circulation

Retricted access

Review article

First published 2012-7

Cystic Fibrosis Transmembrane Conductance Regulator–Modifying Medications: The Future of Cystic Fibrosis Treatment

Rebecca S Pettit

Annals of Pharmacotherapy