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Articles tagged with exon skipping

Exon Skipping

The exon skipping technique tries to change a Duchenne mutation into a Becker mutation. If a variation disturbs the reading frame and thus causes Duchenne dystrophy, the reading frame can be resto...

Tags: exon skipping, exon, antisense oligonucleotide, RNA, duchenne.

Exon Skipping large animal

16th March 2009 Researchers from Children’s National Medical Center and colleagues in Tokyo publish results, video of first successful trial in dogs with Duchenne muscular dystrophy. WASHING...

Tags: exon skipping, dogs, antisense oligonucleotides, molecular patches, morpholinos.

In frame and out of frame deletions

Duchenne and Beckers result from variations on the dystrophin gene.The dystrophin gene is the blueprint, or ‘recipe’ for the body to produce the protein dystrophin.Dystrophin is a protei...

Tags: In frame, out of frame, beckers, exon skipping, stop codon, out of frame deletion.

Exon skipping for Duchenne Muscular Dystrophy: a successful clinical trial in the UK

By Professor Francesco Muntoni, UCL Institute of Child Health LondonProfessor Francesco Muntoni of UCL and Head of the MDEX Consortium discusses the results of the latest successful experimental t...

Tags: Exon skipping, AVI BioPharma, AVI4658, antisense oligonucleotide, exon 51.

Gene therapy research demonstrates promising treatment of DMD for the future

By Matthew WoodMB ChB Cape Town, MA DPhil OxfUniversity of OxfordThe latest announcement of the £150k raised by Action Duchenne to fund the latest research into ‘Exon skipping’, a gene t...

Tags: Exon Skipping, antisense oligonucleotides, peptide nucleic acids, PNA.

Exon Skipping Review April 2009

This review is an excellent way to start undersatnding exon skipping and also to catch up with the very latest developments. You can also view all of Günter Scheuerbrandt’s other excellent r...

Tags: exon skipping, genes, proteins, gene transfer.

Q&A session with Professor Eric Hoffman

Interview with Professor Eric Hoffman on exon skipping in the Duchenne dog model.WECT Channel 6 Wilmington(WECT) - One in every 3,500 baby boys is diagnosed with muscular dystrophy, one of the mos...

Tags: Hoffman, Professor Eric Hoffman, exon skipping, dogs, duchenne dog model.

Dosing regiment

this is potentially important ...Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.http://www.ncbi.nlm.nih.gov/pubmed/194697091: Hu...

Tags: duchenne, dystrophin, dose, peptide, exon skipping, pmo, morpholino.

Leiden University Medical Center and Prosensa B.V. Announce New England Journal of Medicine Publication of First Successful Clinical Study with RNA-based Therapeutic PRO051 in Duchenne Muscular Dystrophy

Novel RNA-based therapeutic PRO051 to enter Phase I/II clinical trials in DMDLeiden, the Netherlands, December 27, 2007. Leiden University Medical Center (LUMC) and Biotech Company Prosensa announ...

Tags: prosensa, exon skipping, clinical trial.

Exon skipping with U7 gene transfer

This is a gene therapy approach for Duchenne muscular dystrophy (DMD), based on the exon-skipping strategy and is currently under development. This approach consists of interacting with the splici...

Tags: gene, DNA, exon skipping, rna, U7, gene transfer.

Reading through premature stop codons with PTC124.

In about 13 to 15% of all Duchenne patients, the disease iscaused by a nonsense mutation in the dystrophin gene. This type of mutation is a single-point change that results in the introduction of ...

Tags: Mutation, exon skipping, dystrophy.

Global excitement about potential treatments for Duchenne

Dr. Matthew Wood reports on recent developments in exon skipping trials from the Oligonucleotide Therapeutic Society meeting held in Fukuoka, Japan in November. Last week I attended the Oligonucle...

Tags: exon skipping, AVI.

Global excitement about potential treatments for Duchenne (Version in French)

Dr. Matthew Wood rapports sur les développements récents dans l’exon skipping essais de la réunion d’oligonucléotides thérapeutiques Société a lieu à Fukuoka, au Japon en Novembre. La ...

Tags: exon skipping, AVI, french.

Exon Skipping - A review by Dr Karl Bettelheim

Article submitted by Dr Karl Bettelheim published on the 3rd of June 2009 IntroductionCurrently there are three major groups of treatment for Duchenne Muscular Dystrophy (DMD) e...

Tags: exon skipping, cellpenetrating peptide CPP, mRNA, antisenseoligonucleotides AOs, Dystrophin.

Phosphorodiamidate morpholino oligomer targeting exon 51.

BackgroundMutations that disrupt the open reading frame and prevent full translation of DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne muscular dystrophy. Olig...

Tags: exon skipping, PMO, morpholino therapy.

Peptide conjugated phosphorodiamidate morpholino oligomer targeting exon 50.

BOTHELL, WA — February 5, 2010 — AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced that it received an orphan drug designation from the Committee for Or...

Tags: PPMO therapy, exon skipping.

2-'O-methyl antisense oligos (PS) targeting exon 44.

BackgroundAntisense-mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allo...

Tags: antisense oligonucleotides, Duchenne muscular dystrophy, exon skipping, 28242Omethyl phosphorothioate, phosphorodiamidate morpholino oligomer.

2-'O-methyl antisense oligos (PS) targeting exon 51.

BACKGROUND:Duchenne’s muscular dystrophy is associated with severe, progressive muscle weakness and typically leads to death between the ages of 20 and 35 years. By inducing specific exon sk...

Tags: myofibers, biopsy, exon skipping, exon 51, prosensa, GSK, clinical trials.

Cocktail morpholinos targeting exon 51 induced highly efficient dystrophin expression.

A promising therapeutic approach for Duchenne muscular dystrophy (DMD) is exon skipping using antisense oligonucleotides (AOs). In-frame deletions of the hinge 3 region of the dystrophin protein, ...

Tags: Exon skipping, muscle pathology.

Systemic Exon 6/8 double skipping demosntrated in dystrophic dogs/human cells.

OBJECTIVE: Duchenne muscular dystrophy (DMD) is caused by the inability to produce dystrophin protein at the myofiber membrane. A method to rescue dystrophin production by antisense oligonucleotid...

Tags: exon skipping, dystrophin.

By-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skipping.

BackgroundDuchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by protein-truncating mutations in the dystrophin gene. Absence of functional dystrophin renders muscle fibr...

Tags: antisense oligomer, B6RosCgDmdmdx4CvJ 4CV mouse, Duchenne muscular dystrophy, exon skipping, morpholino.

Use adeno-associated viral (AAV) vector containing a modified U7 small nuclear [sn] RNA gene

Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by mutations in the dystrophin gene. In most cases, the open-reading frame is disrupted which results in the absence of ...

Tags: Exon skipping, gene trasfer.

Systemic administration of avi-4658, a Phosphorodiamiate morpholino oligomer(pmo) to induce exon 51 skipping, is well tolerated and restores dystrophin expression in duchenne muscular dystrophin (dmd) boys in a dose dependent manner

1) UCL institute of child health, neorology, London,UK2) Royal Victor Infirmary,Newcastle3) 3avi biopharma Bothell USAObjective : we previsiously showed that a single im dose of avi-4658 pmo skips...

Tags: exon skipping, AVI, muscle biopsy, T cells, Phosphorodiamiate morpholino oligomerpmo.

Phsiological charaterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy

Department of cellular and molecular neuroscience, division of neuroscience and mental health, faculty of medicine, imperial college London, Hammersmith hospital campus.Antisense-induced exon skip...

Tags: exon skipping, phophorodiamiate morpholino oligomer PMO, Strength, dystrophin.

Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa guanidine morpholino oligomer treatment.

Myostrain is a negative regulator of muscle mass, and several strategies are being developed to knockdown its expression to improve muscle-wasting conditions. Strategies using antimyostrain-blocki...

Tags: Myostrain, PMO, muscle strength, exon skipping.

Adeno-associated virus for the treatment of musle diseases: Toward Clinical trials.

Muscle diseases include musclular dystrophies, cardiomyopathies, neuromuscular and metabolic disorders. The loss of muscular structure and function is associated with significant morbidity and mor...

Tags: Adenoassociated virus AAV, exon skipping.

Combining stem cells and exon skipping strategy to treat muscular dystrophy

Background: Muscular dystrophies are characterized by primary wasting of skeletal muscle. Mutations in the dystrophin gene cause hereditary muscular diseases such as Becker muscular dystrophy (BMD...

Tags: stem cells, exon skipping, muscular dystrophies.

CPP-directed oligonucleotide exon skipping in animal models of Duchenne muscular dystrophy.

Anatomy and Genetics, Department of Physiology, University of Oxford, South Parks Road, Oxford, UK.Antisense oligonucleotides (AOs) are effective splice switching agents and have potential as ther...

Tags: exon skipping, Antisense oligonucleotides AOs, Duchenne muscular dystrophy DMD.

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is associated with mutations in the dystrophin gene that disrupt the open reading frame whereas the milder Becker’s form is associated with mutations which ...

Tags: Exon Skipping, adenoassociated virus AAV, mRNA, dystrophin gene.

£2.5m research grant for Duchenne Muscular Dystrophy awarded to Oxford researchers

Funding for pioneering research into gene therapy by Action Duchenne has led to further funds for research into this life limiting disease A recent £2.5 million award made by the Health Innovation...

Tags: exon skipping, Duchenne, Duchenne muscular dystrophy, Dr Matthew Wood, Mike Gait, antisense oligonucleotides.

Update on the Therapeutic approaches to muscular dystrophy

This is a summary of the article written by Aurelie Goyenvalle, Jane T Seto, Kay E Davies and Jeffrey Chamberlain, which was published by Oxford University Press on 5 April 2011.The article is a r...

Tags: exon skipping, gene therapy, Viral Vector, Dystrophin, rAAV, Antisense Oligonucleotide, PMO, PPMO, Duchenne, muscular dystrophy, Utrophin.

Exon Skipping Explained

This article by Annemeike Rus is a brilliant explanation of exon skipping.

Tags: exon skipping.