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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
Country/Region
USA
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