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Publications by Felecia Gumienny
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
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Low-Level Dystrophin Expression Attenuating the Dystrophinopathy Phenotype
Muscle and Nerve
Molecular Neuroscience
Neurology
Physiology
Cellular
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
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
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
Personalized Exon Skipping Strategies to Address Clustered Non-Deletion Dystrophin Mutations
Neuromuscular Disorders
Child Health
Neurology
Pediatrics
Perinatology
Genetics
Dual Exon Skipping in Myostatin and Dystrophin for Duchenne Muscular Dystrophy
BMC Medical Genomics
Genetics
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
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Intravenous Infusion of an Antisense Oligonucleotide Results in Exon Skipping in Muscle Dystrophin mRNA of Duchenne Muscular Dystrophy
Pediatric Research
Child Health
Pediatrics
Perinatology
Contemporary Retrotransposition of a Novel Non-Coding Gene Induces Exon-Skipping in Dystrophin mRNA
Journal of Human Genetics
Genetics
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
A Multicenter Comparison of Quantification Methods for Antisense Oligonucleotide-Induced DMD Exon 51 Skipping in Duchenne Muscular Dystrophy Cell Cultures
PLoS ONE
Multidisciplinary