Antisense Oligonucleotides to Restore Dysferlin mRNA and Protein Expression in Dysferlin-deficient Cells
- 詳細技術說明
- 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
- *Abstract
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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
- 國家
- Not Available
- 申請號碼
- None
- 國家/地區
- 美國
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