Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD-Reference-Cited by-同舟云学术

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Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD

Published:2018-12 Issue: Volume:13 Page:534-542
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Akpulat Ugur,Wang Haicui,Becker Kerstin,Contreras Adriana,Partridge Terence A.,Novak James S.,Cirak Sebahattin

Funder

Deutsche Forschungsgemeinschaft

Muscular Dystrophy Association

NIH

International Postdoctoral Research Fellowship Programme of the TUBITAK

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference40 articles.

1. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

2. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals;Koenig;Cell,1987

3. Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort;Flanigan;Hum. Mutat.,2009

4. A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin;Ervasti;J. Cell Biol.,1993

5. Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix;Ibraghimov-Beskrovnaya;Nature,1992

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1. Oligonucleotide therapeutics and their chemical modification strategies for clinical applications;Journal of Pharmaceutical Investigation;2024-02-29

2. Morpholino oligonucleotide-mediated exon skipping for DMD treatment: Past insights, present challenges and future perspectives;Journal of Biosciences;2023-10-11

3. Retinal dystrophins and the retinopathy of Duchenne muscular dystrophy;Progress in Retinal and Eye Research;2022-11

4. A Decade of Progress in Gene Targeted Therapeutic Strategies in Duchenne Muscular Dystrophy: A Systematic Review;Frontiers in Bioengineering and Biotechnology;2022-03-23

5. Restoration of Normal NF1 Function with Antisense Morpholino Treatment of Recurrent Pathogenic Patient-Specific Variant c.1466A>G; p.Y489C;Journal of Personalized Medicine;2021-12-07

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