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Antisense Oligonucleotides to Restore Dysferlin mRNA and Protein Expression in Dysferlin-deficient Cells

Detailed Technology Description: This technology originates from identification of a deep intronic mutation in Dysferlin (DYSF) that alters mRNA splicing to include a mutant peptide fragment within a key DYSF domain. This new antisen

Countries: Not Available

Application No.: None

*Abstract: This technology originates from identification of a deep intronic mutation in Dysferlin (DYSF) that alters mRNA splicing to include a mutant peptide fragment within a key DYSF domain. This new antisense oligo nucleotide(AON-)mediated exon-skipping restores production of normal, full-length DYSF in patients' cells in vitro, offering a novel therapeutic solution for patient with Dysferlin-associated maladies.

*IP Issue Date: None

*IP Type: Other Patent

Country/Region: USA

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