Exon Skipping in development for DMD

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections...

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The Datamonitor Group aims to help its clients operating in the pharmaceuticals, healthcare and biotechnology fields, offering world-leading products and services to meet the challenges of this rapi...

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Labs are the hub of much of modern medicine, either in the hospital setting processing patients blood tests and other samples, in research institutions at many universities or in the headquarters of...

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Sarepta Therapeutics Collaborates For Development Of Addl. Exon-skipping Drug

WASHINGTON (dpa-AFX) - Sarepta Therapeutics (SRPT, AVII) announced a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in development, in sup...

Sarepta to advance second exon-skipping DMD drug into clinic with EU funds

Sarepta Therapeutics and its research partners have capitalized on compelling data for the company's exon-skipping drug eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) with a €5...

Exon skipping to restore gene expression is promising therapeutic strategy for muscular dystrophy

(Mary Ann Liebert, Inc./Genetic Engineering News) A novel therapeutic approach called exon skipping involves bypassing a disease-causing mutation in a gene to restore normal gene expression and protei...

Sarepta Therapeutics and University of Western Australia Announce Exclusive Worldwide Licensing Agreement for Exon-Skipping Program in Duchenne Muscular Dystrophy / Deal Enables Development of New Exon-Skipping Drug Candidates in DMD

CAMBRIDGE, MA and PERTH, AUSTRALIA -- (Marketwired) -- 04/11/13 -- Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and the University of Western Australia (UWA) today announced that they have entered int...

[UWA] licenses exon-skipping drug IP to [AVI BioPharma]

RNAI therapeutics developer [AVI BioPharma Inc.] has received exclusive worldwide rights to the [University of Western Australia]’s patent application, “Antisense Oligonucleotides for Inducing Exo...

Lucky 13 for skipping Sarepta as it nets $118m to progress pipeline

Capitalizing on the enthusiasm for its Duchenne muscular dystrophy (DMD) drug eteplirsen, Sarepta Therapeutics priced an underwritten public offering of common stock on 13 December at $25.25 per share...

A Promising Therapeutic Strategy For Muscular Dystrophy: Exon Skipping To Restore Gene Expression

A novel therapeutic approach called exon skipping involves bypassing a disease-causing mutation in a gene to restore normal gene expression and protein production. Two innovative examples of this stra...

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

Combined Computational-Experimental Analyses of CFTR Exon Strength Uncover Predictability of Exon Skipping Level.

With the increased number of identified nucleotide sequence variations in genes, the current challenge is to classify them as disease-causing or neutral. These variants of unknown clinical significanc...

A missense variant within BRCA1 exon 23 causing exon skipping.

Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations.

Antisense oligomer induced exon skipping is showing promise as a therapy to reduce the severity of Duchenne muscular dystrophy. To date, the focus has been on excluding single exons flanking frame-shi...

CELF proteins regulate CFTR pre-mRNA splicing: essential role of the divergent domain of ETR-3.

Cystic fibrosis is a prominent genetic disease caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Among the many disease-causing alterations are pre-mRNA splic...

RBM5 promotes exon 4 skipping of AID pre-mRNA by competing with the binding of U2AF65 to the polypyrimidine tract.

Alternative splicing is involved in functional regulation of the mutagenic enzyme activation-induced cytidine deaminase (AID). However, the molecular basis for AID splicing regulation remains undefine...