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Publications by Nicolas Wein

60. Intramuscular and Systemic Induction of the N-Truncated Dystrophin by Out-Of-Frame Exon 2 Skipping Restores Muscle Function in the Dup2 Mouse, Providing Further Support for a Therapeutic Pathway for 5’ DMD Mutations

Molecular Therapy

English

Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients’ Cells

Journal of Neuromuscular Diseases

Neurology

2015

English

Related publications

762. Successful Use of Out-Of-Frame Exon 2 Skipping Induces IRES-Driven Expression of the N-Truncated Dystrophin Isoform: Promising Approach for Treating Other 5’ Dystrophin Mutations

English

Antisense-Induced Exon Skipping and Synthesis of Dystrophin in the MDX Mouse

Proceedings of the National Academy of Sciences of the United States of America

Multidisciplinary

2001

English

Personalized Exon Skipping Strategies to Address Clustered Non-Deletion Dystrophin Mutations

Neuromuscular Disorders

English

Dual Exon Skipping in Myostatin and Dystrophin for Duchenne Muscular Dystrophy

BMC Medical Genomics

Genetics

2011

English

134. Assessment of the Dystrophin Gene Exon 53 Skipping Using DMD Patient-Derived Fibroblasts for Exploratory Clinical Trial of Antisense Drug NS-065/NCNP-01

English

Intravenous Infusion of an Antisense Oligonucleotide Results in Exon Skipping in Muscle Dystrophin mRNA of Duchenne Muscular Dystrophy

English

Contemporary Retrotransposition of a Novel Non-Coding Gene Induces Exon-Skipping in Dystrophin mRNA

Journal of Human Genetics

Genetics

2010

English

Splicing Analysis Disclosed a Determinant Single Nucleotide for Exon Skipping Caused by a Novel Intraexonic Four-Nucleotide Deletion in the Dystrophin Gene

Journal of Medical Genetics

Genetics

2006

English

A Multicenter Comparison of Quantification Methods for Antisense Oligonucleotide-Induced DMD Exon 51 Skipping in Duchenne Muscular Dystrophy Cell Cultures

PLoS ONE

Multidisciplinary

2018

English

Amanote Research

Discover open access scientific publications

Search, annotate, share and cite publications

Publications by Nicolas Wein

60. Intramuscular and Systemic Induction of the N-Truncated Dystrophin by Out-Of-Frame Exon 2 Skipping Restores Muscle Function in the Dup2 Mouse, Providing Further Support for a Therapeutic Pathway for 5’ DMD Mutations

Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients’ Cells

Related publications

762. Successful Use of Out-Of-Frame Exon 2 Skipping Induces IRES-Driven Expression of the N-Truncated Dystrophin Isoform: Promising Approach for Treating Other 5’ Dystrophin Mutations

Antisense-Induced Exon Skipping and Synthesis of Dystrophin in the MDX Mouse

Personalized Exon Skipping Strategies to Address Clustered Non-Deletion Dystrophin Mutations

Dual Exon Skipping in Myostatin and Dystrophin for Duchenne Muscular Dystrophy

134. Assessment of the Dystrophin Gene Exon 53 Skipping Using DMD Patient-Derived Fibroblasts for Exploratory Clinical Trial of Antisense Drug NS-065/NCNP-01

Intravenous Infusion of an Antisense Oligonucleotide Results in Exon Skipping in Muscle Dystrophin mRNA of Duchenne Muscular Dystrophy

Contemporary Retrotransposition of a Novel Non-Coding Gene Induces Exon-Skipping in Dystrophin mRNA

Splicing Analysis Disclosed a Determinant Single Nucleotide for Exon Skipping Caused by a Novel Intraexonic Four-Nucleotide Deletion in the Dystrophin Gene

A Multicenter Comparison of Quantification Methods for Antisense Oligonucleotide-Induced DMD Exon 51 Skipping in Duchenne Muscular Dystrophy Cell Cultures