Screening for antisense modulation of dystrophin pre-mRNA splicing
G Dickson, V Hill, IR Graham - Neuromuscular Disorders, 2002 - Elsevier
Most gene therapy approaches to genetic disorders aim to compensate loss-of-function by
introducing recombinant cDNA-based minigenes into diseased tissues. The current report …
introducing recombinant cDNA-based minigenes into diseased tissues. The current report …
The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines …
F Muntoni - Neuromuscular disorders, 2010 - Elsevier
A workshop entitled The Development of Antisense Oligonucleotide Therapies for
Duchenne Muscular Dystrophy, sponsored by the EU funded Clinical Network TREAT-NMD …
Duchenne Muscular Dystrophy, sponsored by the EU funded Clinical Network TREAT-NMD …
Development of exon skipping therapies for Duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues
A Aartsma-Rus, V Straub, R Hemmings… - Nucleic acid …, 2017 - liebertpub.com
Duchenne muscular dystrophy (DMD) is a rare, severe, progressive muscle-wasting disease
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …
Antisense oligonucleotides: A primer
There are few disease-modifying therapeutics for neurodegenerative diseases, but
successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal …
successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal …
Exon‐skipping therapy for Duchenne muscular dystrophy
A Nakamura, S Takeda - Neuropathology, 2009 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is a lethal muscle disorder caused by mutations in the
DMD gene for which no mutation‐targeted therapy has been available thus far. However …
DMD gene for which no mutation‐targeted therapy has been available thus far. However …
A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping
T Shimo, K Hosoki, Y Nakatsuji, T Yokota… - Journal of human …, 2018 - nature.com
Oligonucleotide-mediated splicing modulation is a promising therapeutic approach for
Duchenne muscular dystrophy (DMD). Recently, eteplirsen, a phosphorodiamidate …
Duchenne muscular dystrophy (DMD). Recently, eteplirsen, a phosphorodiamidate …
Modulation of RNA splicing by oligonucleotides: Mechanisms of action and therapeutic implications
OV Sergeeva, EY Shcherbinina, N Shomron… - nucleic acid …, 2022 - liebertpub.com
Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic
approaches have been developed to correct aberrant alternative splicing events for the …
approaches have been developed to correct aberrant alternative splicing events for the …
Antisense oligonucleotide-based therapy for neuromuscular disease
Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular
Atrophy are neurodegenerative genetic diseases characterized primarily by muscle …
Atrophy are neurodegenerative genetic diseases characterized primarily by muscle …
Therapeutic antisense oligonucleotides for movement disorders
E Doxakis - Medicinal research reviews, 2021 - Wiley Online Library
Movement disorders are a group of neurological conditions characterized by abnormalities
of movement and posture. They are broadly divided into akinetic and hyperkinetic …
of movement and posture. They are broadly divided into akinetic and hyperkinetic …
New insights in gene‐derived therapy: the example of Duchenne muscular dystrophy
A Aartsma‐Rus, JT den Dunnen… - Annals of the New …, 2010 - Wiley Online Library
The two therapeutic approaches currently most advanced in clinical trials for Duchenne
muscular dystrophy are antisense‐mediated exon skipping and forced read‐through of …
muscular dystrophy are antisense‐mediated exon skipping and forced read‐through of …