Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study-Reference-Cited by-同舟云学术

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Published:2011-08 Issue:9791 Volume:378 Page:595-605
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Cirak Sebahattin,Arechavala-Gomeza Virginia,Guglieri Michela,Feng Lucy,Torelli Silvia,Anthony Karen,Abbs Stephen,Garralda Maria Elena,Bourke John,Wells Dominic J,Dickson George,Wood Matthew JA,Wilton Steve D,Straub Volker,Kole Ryszard,Shrewsbury Stephen B,Sewry Caroline,Morgan Jennifer E,Bushby Kate,Muntoni Francesco

Funder

Great Ormond Street Hospital Children's Charity. Newcastle University, UCL, and Oxford are partners in TREAT-NMD

Publisher

Elsevier BV

Subject

General Medicine

Reference34 articles.

1. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management;Bushby;Lancet Neurol,2010

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

3. The clinical, genetic and dystrophin characteristics of Becker muscular dystrophy. II. Correlation of phenotype with genetic and protein abnormalities;Bushby;J Neurol,1993

4. Splice switching oligonucleotides as potential therapeutics;Sazani,2008

5. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles;Lu;Proc Natl Acad Sci USA,2005

Cited by 750 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Enhancing peptide and PMO delivery to mouse airway epithelia by chemical conjugation with the amphiphilic peptide S10;Molecular Therapy - Nucleic Acids;2024-09

2. Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping;Molecular Therapy - Nucleic Acids;2024-09

3. Phase I/II Trial of Brogidirsen: Dual-Targeting Antisense Oligonucleotides for Exon 44 Skipping in Duchenne Muscular Dystrophy;2024-08-30

4. Therapeutic Antisense Oligonucleotides in Oncology: From Bench to Bedside;Cancers;2024-08-23

5. Tailored antisense oligonucleotides designed to correct aberrant splicing reveal actionable groups of mutations for rare genetic disorders;Experimental & Molecular Medicine;2024-08-01