Antisense Oligonucleotides to Restore Dysferlin mRNA and Protein Expression in Dysferlin-deficient Cells
- 详细技术说明
- 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
-
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
- 国家
- Not Available
- 申请号码
- None
- 国家/地区
- 美国
欲了解更多信息，请点击 这里
