Abstract: Compositions and methods for the treatment of asthma with oligonucleotides which specifically hybridize with nucleic acids encoding B7 proteins.
Abstract: This invention relates to compounds, compositions, and methods useful for modulating XIAP gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of XIAP gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of XIAP genes.
Abstract: Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene. Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations. The invention also includes small interfering RNAs (siRNAs) and small hairpin RNAs (shRNAs) that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS). The present invention further provides asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.
Abstract: A therapeutic and/or cosmetic formulation comprising at least one anti-sense polynucleotide to a connexin protein together with a pharmaceutically acceptable carrier or vehicle is useful in site specific down regulation of connexin protein expression, particularly in reduction of neuronal cells death, wound healing, reduction of inflammation, decrease of scar formation and skin rejuvenation and thickening.
Type:
Grant
Filed:
August 25, 2006
Date of Patent:
February 1, 2011
Assignee:
Coda Therapeutics, Inc.
Inventors:
Richard Colin Green, David Laurence Becker
Abstract: Compounds, compositions and methods are provided for modulating the expression of C-reactive protein. The compositions comprise oligonucleotides, targeted to nucleic acid encoding C-reactive protein. Methods of using these compounds for modulation of C-reactive protein expression and for diagnosis and treatment of disease associated with expression of C-reactive protein are provided.
Abstract: RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.
Type:
Grant
Filed:
October 26, 2007
Date of Patent:
December 28, 2010
Assignee:
Duke University
Inventors:
Bruce A. Sullenger, Christopher P. Rusconi
Abstract: A pharmaceutical composition blocks angiogenesis and contains as an active agent at least one nucleotide sequence from nucleic acid molecule SEQ ID NO. 3, fragments thereof containing at least twelve contiguous nucleotides and derivatives thereof; and nucleic acid sequences containing at least twelve contiguous nucleotides of the nucleic acid molecule SEQ ID NO 30 and derivatives thereof.
Abstract: This invention provides methods of inducing apoptosis in a bcl-6-expressing cell and methods of treating a subject with a cancer comprising a bcl-6-expressing cell, comprising administration of a composition that reduces the amount of the bcl-6 protein or of an mRNA molecule encoding same, a composition comprising a nucleic acid molecule complementary to or corresponding to a region of the mRNA molecule, or a vector expressing the nucleic acid molecule. In another embodiment, the present invention provides an isolated nucleic acid molecule having a sequence corresponding to or complementary to accessible regions of bcl-6 mRNA, and vectors, cells, compositions, and kits comprising same.
Type:
Grant
Filed:
March 22, 2005
Date of Patent:
December 14, 2010
Assignee:
The Trustees of the University of Pennsylvania
Abstract: The present invention is directed to methods for enhancing the replicative capacity of cells, by culturing the cells in the presence of an active agent or compound which inhibits SIRT1. One method provides expanding stem cells by culturing the cells in the presence of a SIRT1 inhibitor. The resulting cultured cells can be used for a variety of applications including cell-based therapies such as bone marrow transplants, gene therapies, tissue engineering, and in vitro organogenesis.
Type:
Grant
Filed:
June 14, 2006
Date of Patent:
November 23, 2010
Assignee:
Children's Medical Center Corporation
Inventors:
Frederick W. Alt, David B. Lombard, Katrin F. Chua, Raul Mostoslavsky, Hwei-Ling Cheng
Abstract: Translation of the hepatitis C virus (HCV) RNA is mediated by the interaction of ribosomes and cellular proteins with an internal ribosome entry site (IRES) located within the 5?untranslated region (5?UTR). We have investigated whether small RNA molecules corresponding to the different stem-loop (SL) domains of the HCV IRES, when introduced in trans, can bind to the cellular proteins and antagonize their binding to the viral IRES, thereby inhibiting HCV IRES-mediated translation. We have found that an RNA molecule corresponding to SL III of the HCV IRES could efficiently inhibit HCV IRES-mediated translation in a dose-dependent manner without affecting cap-dependent translation. The SL III RNA was also found to bind efficiently to most of the cellular proteins which interacted with the HCV 5?UTR. A smaller RNA corresponding to SL e+f of domain III also strongly and selectively inhibited HCV IRES-mediated translation.
Abstract: An improved understanding and method for the clinical adjuvant and immunomodulatory use of dsRNAs and ply-ICLC in particular, alone or in conjunction with other drugs and various vaccines designed to prevent or treat various microbial, viral, neoplastic, autoimmune diseases, and or degenerative diseases.
Abstract: In a first aspect thereof, the present invention provides a method for treatment and/or prevention of a disease selected from the group consisting of psoriasis and squamous cell carcinoma by inhibiting the expression of squamous cell carcinoma antigen (SCCA) by cells. In another aspect thereof, the present invention provides a method for screening for substances that inhibit epidermal parakeratosis, wherein the activity of a candidate substance that inhibits cysteine protease inhibitory activity possessed by squamous cell carcinoma antigen 1 (SCCA-1) is used as an indicator.
Abstract: Methods for producing interfering RNA molecules in mammalian cells are provided. Therapeutic uses for the expressed molecules, including inhibiting expression of HIV, are also provided.
Abstract: Antisense compounds, compositions and methods are provided for modulating the expression of hydroxysteroid 11-beta dehydrogenase 1. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding hydroxysteroid 11-beta dehydrogenase 1. Methods of using these compounds for modulation of hydroxysteroid 11-beta dehydrogenase 1 expression and for treatment of diseases associated with expression of hydroxysteroid 11-beta dehydrogenase 1 are provided.
Abstract: RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.
Type:
Grant
Filed:
October 26, 2007
Date of Patent:
October 12, 2010
Assignee:
Duke University
Inventors:
Bruce A. Sullenger, Christopher P. Rusconi
Abstract: Compounds, compositions and methods are provided for modulating the expression of eIF4E-BP2. The compositions comprise oligonucleotides, targeted to nucleic acid encoding eIF4E-BP2. Methods of using these compounds for modulation of eIF4E-BP2 expression and for diagnosis and treatment of diseases and conditions associated with expression of eIF4E-BP2 are provided.
Type:
Grant
Filed:
November 21, 2006
Date of Patent:
October 5, 2010
Assignee:
Isis Pharmaceuticals, Inc.
Inventors:
Sanjay Bhanot, Kenneth W. Dobie, Ravi Jain
Abstract: Compounds, compositions and methods are provided for modulating the expression of HIF1-beta. The compositions comprise oligonucleotides, targeted to nucleic acid encoding HIF1-beta. Methods of using these compounds for modulation of HIF1-beta expression and for diagnosis and treatment of diseases and conditions associated with expression of HIF1-beta are provided.
Type:
Grant
Filed:
October 13, 2009
Date of Patent:
September 21, 2010
Assignee:
Isis Pharmaceuticals, Inc.
Inventors:
Eric G. Marcusson, Scott W. Henry, Youngsoo Kim, Kenneth Dobie
Abstract: Nucleic acids and vectors for interfering with the expression of membrane efflux transport proteins in cells that express such proteins are provided. Also provided are cells and cell lines comprising such nucleic acids and vectors. Methods for screening chemicals and biomolecules for gastrointestinal absorption in animals, and kits for practicing such methods are also provided.
Type:
Grant
Filed:
November 9, 2007
Date of Patent:
September 14, 2010
Assignee:
Absorption Systems Group, LLC
Inventors:
Albert J. Owen, III, Ismael J. Hidalgo, Jibin Li, Wei Zhang
Abstract: An antibacterial antisense conjugate and method of using the same for treating a bacterial infection in a mammalian host are disclosed. The conjugate includes an antisense oligonucleotide conjugated to a carrier peptide that significantly enhances the antibacterial activity of the oligonucleotide. The antisense oligonucleotide contains 10-20 nucleotide bases and has a targeting nucleic acid sequence complementary to a target sequence containing or within 10 bases, in a downstream direction, of the translational start codon of a bacterial mRNA that encodes a bacterial protein essential for bacterial replication, where the compound binds to a target mRNA with a Tm of between 50° to 60° C. The carrier peptide is an arginine-rich peptide containing between 6 and 12 amino acids.
Type:
Grant
Filed:
July 13, 2006
Date of Patent:
September 7, 2010
Assignee:
AVI BioPharma Inc.
Inventors:
Bruce L. Geller, Patrick L. Iversen, Lucas D. Tilley
Abstract: RNA aptamers and methods for identifying the same are disclosed. The RNA aptamers selectively bind coagulation factors, E2F family members, Ang1 or Ang2, and therapeutic and other uses for the RNA aptamers are also disclosed.
Type:
Grant
Filed:
October 26, 2007
Date of Patent:
August 17, 2010
Assignee:
Duke University
Inventors:
Bruce A. Sullenger, Christopher P. Rusconi, Rebekah R. White