Article

Structural Perturbations of Exon Skipping Edits within the Dystrophin D20:24 Region

2020-10-11

Abstract excerpt

Exon skipping is a disease modifying therapy that operates at the RNA level. In this strategy, oligonucleotide analog drugs are used to specifically mask specific exons and prevent them from inclusion in the mature mRNA. Of course, this also results in loss of the corresponding region from the cognate protein, which is one possible therapeutic aim. Exon skipping can also be used to restore protein expression in ca...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: cd3f00da-656a-55fc-a82a-da745069fa31
DOI: 10.1101/2020.10.10.334763

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Structural Perturbations of Exon Skipping Edits within the Dystrophin D20:24 RegionDOI 10.1101/2020.10.10.334763Read

Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and ChallengesSemantic proximity Read

Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.Semantic proximity Read

Exon-Skipping in Duchenne Muscular Dystrophy.Semantic proximity Read

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy.Semantic proximity Read

Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation.Semantic proximity Read

Multiple exon skipping and RNA circularisation contribute to the severe phenotypic expression of exon 5 dystrophin deletion.Semantic proximity Read

Empirical and Computational Comparison of Alternative Therapeutic Exon Skip Repairs for Duchenne Muscular DystrophySemantic proximity Read

Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?Semantic proximity Read

An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database.Semantic proximity Read

Therapeutic approaches for Duchenne muscular dystrophy.Semantic proximity Read

Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular AtrophySemantic proximity Read

Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approachSemantic proximity Read