[HTML][HTML] Quantitative antisense screening and optimization for exon 51 skipping in Duchenne muscular dystrophy
Y Echigoya, KRQ Lim, N Trieu, B Bao, BM Nichols… - Molecular Therapy, 2017 - cell.com
Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused
by mutations in the dystrophin (DMD) gene. Exon skipping is a therapeutic approach that …
by mutations in the dystrophin (DMD) gene. Exon skipping is a therapeutic approach that …
Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?
Introduction: Duchenne muscular dystrophy (DMD), one of the most common and lethal
genetic disorders, is caused by mutations of the dystrophin gene. Removal of an exon or of …
genetic disorders, is caused by mutations of the dystrophin gene. Removal of an exon or of …
Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites
A Aartsma-Rus, CL De Winter, AAM Janson… - …, 2005 - liebertpub.com
As small molecule drugs for Duchenne muscular dystrophy (DMD), antisense
oligonucleotides (AONs) have been shown to restore the disrupted reading frame of DMD …
oligonucleotides (AONs) have been shown to restore the disrupted reading frame of DMD …
Therapeutic Modulation of DMD Splicing by Blocking Exonic Splicing Enhancer Sites with Antisense Oligonucleotides
A AARTSMA‐RUS, AAM Janson… - Annals of the New …, 2006 - Wiley Online Library
Antisense oligonucleotides (AONs) can be used to correct the disrupted reading frame of
Duchenne muscular dystophy patients (DMD). We have a collection of 121 AONs, of which …
Duchenne muscular dystophy patients (DMD). We have a collection of 121 AONs, of which …
[HTML][HTML] Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching
CT Fragall, AM Adams, RD Johnsen, R Kole… - BMC Medical …, 2011 - Springer
Background Antisense oligomer induced exon skipping aims to reduce the severity of
Duchenne muscular dystrophy by redirecting splicing during pre-RNA processing such that …
Duchenne muscular dystrophy by redirecting splicing during pre-RNA processing such that …
Targeting RNA to treat neuromuscular disease
F Muntoni, MJA Wood - Nature reviews Drug discovery, 2011 - nature.com
The development of effective therapies for neuromuscular disorders such as Duchenne
muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the …
muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the …
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
G McClorey, HM Moulton, PL Iversen, S Fletcher… - Gene therapy, 2006 - nature.com
Manipulation of pre-mRNA splicing by antisense oligonucleotides (AOs) offers considerable
potential for a number of genetic disorders. One of these is Duchenne muscular dystrophy …
potential for a number of genetic disorders. One of these is Duchenne muscular dystrophy …
[HTML][HTML] Long-term exon skipping studies with 2′-O-methyl phosphorothioate antisense oligonucleotides in dystrophic mouse models
CL Tanganyika-de Winter, H Heemskerk… - … Therapy-Nucleic Acids, 2012 - cell.com
Antisense-mediated exon skipping for Duchenne muscular dystrophy (DMD) is currently
tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping …
tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping …
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy
G Filonova, A Aartsma-Rus - Expert Opinion on Biological Therapy, 2023 - Taylor & Francis
Introduction It is established that the exon-skipping approach can restore dystrophin in
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …
Exon-skipping advances for Duchenne muscular dystrophy
L Echevarría, P Aupy… - Human molecular genetics, 2018 - academic.oup.com
Duchenne muscular dystrophy (DMD) is a fatal genetic disorder characterized by
progressive muscle wasting that has currently no cure. Exon-skipping strategy represents …
progressive muscle wasting that has currently no cure. Exon-skipping strategy represents …