Abstract: The present invention relates to the prevention and/or treatment of retinal dystrophy in a patient, including Leber congenital amaurosis (LCA).
Abstract: The present disclosure provides antisense oligonucleotides targeting Regulator of telomere elongation helicase 1 (RTEL1). The disclosure also provides, enhanced antisense oligonucleotides targeting RTEL1 for use in treating and/or preventing a hepatitis B virus (HBV) infection. Also disclosed are pharmaceutical compositions and their use.
Type:
Grant
Filed:
February 1, 2022
Date of Patent:
February 13, 2024
Assignee:
Hoffmann-La Roche Inc.
Inventors:
Erik Funder, Natascha Hruschka, Susanne Kammler, Erich Koller, Brian Leonard, Souphalone Luangsay, Susanne Mohr, Tobias Nilsson, Søren Ottosen, Lykke Pedersen, Søren V. Rasmussen, Steffen Schmidt, Sabine Sewing, Daniel Turley, Johanna Marie Walther
Abstract: Disclosed is an oligonucleotide or a pharmacologically acceptable salt thereof, wherein the oligonucleotide comprises at least one defined nucleoside structure, can bind to a human nSR100 gene and has human nSR100 expression inhibiting activity. The oligonucleotide has a length of 12 to 20 mer, and is complementary to a defined target region. Further, disclosed is an nSR100 gene expression inhibitor and a cancer therapeutic agent containing the oligonucleotide or the pharmacologically acceptable salt thereof. The cancer therapeutic agent is used for treatment of small cell lung cancer, prostate cancer, or breast cancer.
Type:
Grant
Filed:
July 31, 2019
Date of Patent:
January 9, 2024
Assignees:
Osaka Univerity, National Institutes of Biomedical Innovation, Health and Nutrition, Luxna Biotech Co., Ltd.
Abstract: Systems and method for providing chaperone activity to a protein-containing compound is disclosed. The method includes selecting a nucleic acid based on one or more of the nucleic acid's particular properties and a specific sequence of the nucleic acid and applying the nucleic acid to a compound comprising one or more proteins to provide chaperone activity to the compound.
Type:
Grant
Filed:
November 16, 2020
Date of Patent:
October 10, 2023
Assignee:
University of Denver
Inventors:
Scott Horowitz, Adam Begeman, Ahhyun Son, Alexa Gomez, Theodore Litberg