THIOMORPHOLINO ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF PTP1B RELATED DISEASES

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 ASO...

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Hauptverfasser:	KOSTOV, Ondrej, SUDHEENDRAN, Kavitha, VEEDU, Rakesh, LE, Tri, CARUTHERS, Marvin, JASTRZEBSKA, Katarzyna, SCHAFER, Balazs
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	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. La présente invention concerne des oligonucléotides antisens (ASOs) utilisés pour traiter, prévenir ou améliorer la progression d'états tels que le diabète sucré de Type 2 (T2DM) et la résistance à l'insuline, la résistance à la leptine et l'obésité, le syndrome de Rett et les cancers. Spécifiquement, les ASOs contenant thiomorpholino ciblent et induisent un saut d'exon (y compris l'exon 2) du transcrit de gène de protéine tyrosine phosphatase non-récepteur de Type 1 ( PTPN1) pendant le traitement d'ARN, ce qui permet d'inhiber l'expression de la protéine tyrosine phosphatase-1B (PTP1B).