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
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creator KOSTOV, Ondrej
SUDHEENDRAN, Kavitha
VEEDU, Rakesh
LE, Tri
CARUTHERS, Marvin
JASTRZEBSKA, Katarzyna
SCHAFER, Balazs
description 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).
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subjects 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
title THIOMORPHOLINO ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF PTP1B RELATED DISEASES
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