ReviewEuropean journal of human genetics : EJHG2010
Progress in therapeutic antisense applications for neuromuscular disorders.
Review in European journal of human genetics : EJHG, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
18 citing papers in PubMed, 36 citations in OpenAlex.
- Physiological and druggable skipping of immunoglobulin variable exons in plasma cells.Cellular & molecular immunology · 2019Article
- Mechanisms of Antisense Transcription Initiation with Implications in Gene Expression, Genomic Integrity and Disease Pathogenesis.Non-coding RNA · 2019Review
- Characteristics of Antisense Transcript Promoters and the Regulation of Their Activity.International journal of molecular sciences · 2015Review
- Circulating Muscle-specific miRNAs in Duchenne Muscular Dystrophy Patients.Molecular therapy. Nucleic acids · 2014Article
- Electrical impedance myography for the in vivo and ex vivo assessment of muscular dystrophy (mdx) mouse muscle.Muscle & nerve · 2014Article
- Mechanisms of antisense transcription initiation from the 3' end of the GAL10 coding sequence in vivo.Molecular and cellular biology · 2013Article
- Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models.Molecular therapy. Nucleic acids · 2012Article
- Efficient in vitro siRNA delivery and intramuscular gene silencing using PEG-modified PAMAM dendrimers.Molecular pharmaceutics · 2012Article
- Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis.Cellular and molecular life sciences : CMLS · 2012Review
- Discovery, synthesis, and biological evaluation of novel SMN protein modulators.Journal of medicinal chemistry · 2011Article
- Therapeutics development in myotonic dystrophy type 1.Muscle & nerve · 2011Review
- miRNAs as serum biomarkers for Duchenne muscular dystrophy.EMBO molecular medicine · 2011Article
- Therapeutic exon 'switching' for dysferlinopathies?European journal of human genetics : EJHG · 2010Article
- Therapy development in spinal muscular atrophy.Nature neuroscience · 2010Article
- RNA splicing meets genetic testing: detection and interpretation of splicing defects in genetic diseases.European journal of human genetics : EJHG · 2010Article
- Expression of dog microdystrophin in mouse and dog muscles by gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Therapeutic developments in spinal muscular atrophy.Therapeutic advances in neurological disorders · 2010Article
- Myotonia congenita and myotonic dystrophy: surveillance and management.Current treatment options in neurology · 2010Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Neuromuscular disorders are a frequent cause of chronic disability in man. They often result from mutations in single genes and are thus, in principle, well suited for gene therapy. However, the tissues involved (muscle and the central nervous system) are post-mitotic, which poses a challenge for most viral vectors. In some cases, alternative approaches may use small molecules, for example, antisense oligonucleotides (AONs). These do not deliver a new gene, but rather modulate existing gene products or alter the utilization of pathways. For Duchenne muscular dystrophy, this approach is in early phase clinical trials, and for two other common neuromuscular disorders (spinal muscular atrophy and myotonic dystrophy), significant preclinical advances have recently been made.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.