October 2017

BNA Gapmer Reverts Splicing in MDCs

Bio-synthesis Inc. is pleased to announce a new publication regarding Bridged Nucleic Acids: "BNA Gapmers revert splicing and reduce RNA foci" by Manning et al. at Baylor College of Medicine, Houston, Texas.

BNA gapmers revert splicing defects in myotonic dystrophy type 1 (DM1) cells. DM1 is a multi-systemic disease caused by an expanded CTG repeat in the 3'-untranslated region (UTR) of the dystrophia myotonica protein kinase (DMPK) gene. Myotonic dystrophy is the most common type of muscular dystrophy. In DM1 the expanded CUG repeat RNA (CUGexp RNA) is retained in the nucleus where it forms RNA foci which lead to defects in regulated alternative splicing events during development.

Manning et al. recently used an antisense BNA gapmer strategy to degrade CUGexp RNA in immortalized human TeloMyoD fibroblast cell lines expressing telomerase and containing a tetracycline-inducible MyoD to promote the myogenic program in response to cell growth. FISH allowed visualization of the RNA foci in both untreated and treated cells. BNA gapmers targeting within the repetitive region of DMPK preferentially degrade the mutant allele thereby decreasing RNA foci.

You may read the full publication at https://www.biosyn.com/pdf/BNA-nc-gapmer-revert-splicing.pdf

We hope you find this publication a valuable resource and invite you to contact us or visit our web site to learn more about Bridged Nucleic Acids as referenced by the links below:

References: Kassie S. Manning, Ashish N. Rao, Miguel Castro, Thomas A. C

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