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AssigneesThe Regents of the University of Colorado, a body corporate, Murdoch University
InventorsMarvin H Caruthers, Rakesh Naduvile Veedu, Tri Bao Le, Balazs Schafer, Ondrej Kostov, Kavitha Sudheendran, Katarzyna Jastrzebska, Mathias Danielsen, Mariia Ciba, Thomas Lasek
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The present invention relates to antisense oligonucleotides (ASOs) used to treat, prevent or ameliorate the progression of conditions such as Type 2 Diabetes Mellitus (T2DM) and insulin resistance, leptin resistance and obesity, Rett syndrome, and cancers. Specifically, thiomorpholino-containing ASOs target and induce exon skipping (including exon 2) of the Protein Tyrosine Phosphatase Non-Receptor Type 1 (PTPN1) gene transcript during RNA processing, thereby inhibiting the expression of protein tyrosine phosphatase-1B (PTP1B) protein.
An isolated or purified antisense oligomer targeted to a nucleic acid molecule encoding human Protein Tyrosine Phosphatase Non-Receptor Type 1 (PTPN1) pre-mRNA, wherein the antisense oligomer has a nucleobase sequence selected from:
(canceled)
20-23. (canceled)
24-49. (canceled)
Dependencies are inferred from the claim text.