Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF4 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with IRF4.
Type:
Grant
Filed:
March 1, 2019
Date of Patent:
February 8, 2022
Assignee:
Ionis Pharmaceuticals, Inc.
Inventors:
Tianyuan Zhou, Youngsoo Kim, Robert MacLeod, Huynh-Hoa Bui, Susan M. Freier
Abstract: Compositions are directed to BCL2-associated athanogene 3 (BAG3) molecules and agents which modulate expression of BAG3 molecules. Pharmaceutical composition for administration to patients, for example, patients with heart failure, comprise one or more BAG3 molecules or agents which modulate expression of BAG3. Methods of treatment and identifying candidate therapeutic agents are also provided.
Type:
Grant
Filed:
January 30, 2015
Date of Patent:
February 1, 2022
Assignee:
TEMPLE UNIVERSITY OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
Inventors:
Arthur M. Feldman, Douglas G. Tilley, Weizhong Zhu, Kamel Khalili, Walter J. Koch
Abstract: The present invention discloses a set of human microRNAs, or a primary transcript for such microRNAs, or a precursor of such microRNAs, or a mimic of such microRNAs or a combination thereof, and their use as medicaments for inducing proliferation of cardiomyocytes for the prevention and treatment of heart diseases associated with a loss of cardiomyocytes. The invention also relates to a method for screening microRNAs and biological and therapeutically active compounds for their ability to increase proliferation of cardiomyocytes.
Type:
Grant
Filed:
May 1, 2019
Date of Patent:
February 1, 2022
Assignee:
King's College London
Inventors:
Mauro Giacca, Serena Zacchigna, Miguel Luis Cunha Mano, Ana Sofia Bregieiro Eulalio
Abstract: The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the serum amyloid P component (APCS) gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of an APCS gene and to treat subjects having an APCS-associated disease, e.g., amyloidosis, Alzheimer's disease or coronary atherosclerotic heart disease.
Abstract: The present invention relates to exosomes, loaded with genetic material and methods of producing them and to the use of such exosomes for delivering genetic material in vivo, in particular the use of such exosomes in methods of gene therapy or gene silencing.
Abstract: Method for controlling for the appearance of seizures in the mammalian brain comprising modifying the abundance of a specific miRNA—miR-211, for uses in preventing seizures and providing a model system to examine the effect of a drug or a treatment to seizures.
Abstract: This invention relates to compounds, compositions, and methods useful for reducing ?-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Abstract: In some aspects, the disclosure relates to methods and compositions for modulation of miR-122 expression in a cell or a subject. In some embodiments, methods and compositions described by the disclosure are useful for the treatment of liver-associated diseases (e.g., chronic liver disease, alcoholic liver disease).
Abstract: The present invention refers to an oligonucleotide which is an effective inhibitor of microRNA miR-132 and its use in medicine, particularly in the prevention or treatment of cardiac and/or fibrotic disorders.
Type:
Grant
Filed:
December 14, 2018
Date of Patent:
December 28, 2021
Assignee:
Medizinische Hochschule Hannover
Inventors:
Thomas Thum, Sandor Batkai, Ariana Foinquinos
Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.
Type:
Grant
Filed:
September 4, 2018
Date of Patent:
December 14, 2021
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
David Bumcrot, Brian Bettencourt, Ivanka Toudjarska
Abstract: Melanophilin (MLPH) of the present invention is involved in differentiation into an adipocyte or fat accumulation, and accordingly, obesity can be treated or prevented by inhibiting the MLPH. Further, by measuring an expression level of the MLPH, obesity can be diagnosed and treated, and therapeutic agents for obesity and agents regulating differentiation into adipocytes can be screened.
Type:
Grant
Filed:
August 18, 2016
Date of Patent:
November 30, 2021
Assignee:
KOREA INSTITUTE OF RADIOLOGICAL & MEDICAL SCIENCES
Inventors:
Kee Ho Lee, Yang Hyun Kim, Eun Ran Park, Sung Sub Kim, Hyun Jin Shin, Eun Ju Lee, Chun Ho Kim, Sang Jun Park, Mi-yeun Kim, Jie Young Song
Abstract: The present invention relates to a composition for producing an extracellular matrix from a eukaryotic cell, the composition comprising a polypeptide or compound capable of specifically binding to a microtubule associated serine/threonine kinase family member 4 (MAST4) protein or a fragment thereof or a polynucleotide, polypeptide or compound capable of specifically binding to a nucleic acid coding for the MAST4 protein or a fragment thereof, and a composition for promoting chondrogenesis, comprising the same composition.
Type:
Grant
Filed:
March 8, 2018
Date of Patent:
November 23, 2021
Inventors:
Seong Jin Kim, Saerom Kim, Yeung Won June Kim, Ypsse Kim
Abstract: Described herein are methods and compositions relating to the treatment of cancer, e.g., breast cancer, using, e.g., aptamer-siRNA chimera molecules.
Type:
Grant
Filed:
May 10, 2019
Date of Patent:
November 23, 2021
Assignee:
CHILDREN'S MEDICAL CENTER CORPORATION
Inventors:
Judy Lieberman, Adi Gilboa-Geffen, Lee Adam Wheeler
Abstract: RNA from a biological fluid is stabilized during isolation and/or storage using DNA. In especially preferred aspects, the RNA is cfRNA and/or ctRNA, and the biological fluid is blood.
Type:
Grant
Filed:
May 30, 2018
Date of Patent:
November 9, 2021
Assignee:
NantOmics LLC
Inventors:
Patrick Soon-Shiong, Shahrooz Rabizadeh, Kathleen Danenberg
Abstract: This disclosure relates to methods and compositions for managing vascular conditions by targeting microRNA. In certain embodiments, the disclosure relates to antisense, RNA interference, and blocking oligonucleotide therapeutic compositions and uses related thereto.
Type:
Grant
Filed:
December 3, 2019
Date of Patent:
October 19, 2021
Assignees:
Emory University, Georgia Tech Research Corporation
Inventors:
Hanjoong Jo, Dongju Son, Wakako Takabe, Sandeep Kumar, Haiwei Qiu, Chanwoo Kim
Abstract: The present invention relates to methods to treat or prevent cancers in a subject, in particular the present invention relates to a method of treating and/or preventing cancer comprising targeting cancer stem cells by administering miRNAs which have reduced expression or are lacking in the cancer stem cells. in some embodiments, the miRNAs that are reduced or lacking in cancer stem cells are let-7 miRNAs. In alternative embodiments, the present invention relates to a method of treating and/or preventing cancer comprising targeting cancer stem cells by administering miRNAs which have increased expression levels in the cancer stem cells. Another aspect of the present invention relates to methods to enrich for a cancer stem cell population. Another aspect of the present invention relates to methods to identify miRNAs which contribute to the self-renewal capacity of cancer stem cells.
Type:
Grant
Filed:
December 6, 2018
Date of Patent:
October 19, 2021
Assignee:
Children's Medical Center Corporation
Inventors:
Judy Lieberman, Erwei Song, Fengyan Yu, Xiaoqu Hu
Abstract: The present invention provides a single-stranded nucleic acid molecule having a delivery function and capable of inhibiting expression of a target gene. The single-stranded nucleic acid molecule of the present invention is a single-stranded nucleic acid molecule composed of a region (Xc), a linker region (Lx) and a region (X), wherein said region (Xc) is complementary to said region (X), at least one of said region (X) and said region (Xc) contains an expression inhibitory sequence that inhibits expression of the target gene, and a bio-related substance having a delivery function is bonded to at least one selected from the group consisting of the 5?-terminus, the 3?-terminus, and said linker region (Lx).
Abstract: The present invention relates to antisense oligonucleotides for modulating the activity of PRDM15 and use thereof in the treatment of cancer. In particular, said antisense oligonucleotides are capable of inducing the skipping of an exon of a PRDM15 mRNA. The present invention also relates to a method for determining prognosis in a patient with cancer, or selecting a therapeutic strategy for a patient with cancer, by assessing the level of PRDM15 nucleic acid, protein or activity in a sample.
Type:
Grant
Filed:
September 4, 2017
Date of Patent:
October 5, 2021
Assignee:
AGENCY FOR SCIENCE, TECHNOLOGY AND RESEARCH
Abstract: The present disclosure provides methods and compositions for the treatment of cancer. In some aspects, the present disclosure provides splice-switching oligonucleotides that downregulate AR or EGFR expression and methods of using these splice-switching oligonucleotides to treat cancer.
Type:
Grant
Filed:
November 4, 2016
Date of Patent:
October 5, 2021
Assignee:
Duke University
Inventors:
Jennifer Freedman, Brendon Patierno, Bonnie Lacroix, Timothy Robinson, Bruce Sullenger, Daniel George, Steven Patierno