Abstract: The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing BCL2 gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-over-lapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a BCL2 mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a BCL2 gene in a cell or in a subject to treat a BCL2-related disease.
Type:
Application
Filed:
February 29, 2008
Publication date:
February 18, 2010
Applicant:
MDRNA, INC.
Inventors:
Steven C. Quay, James McSwiggen, Narendra K. Vaish, Mohammad Ahmadian
Abstract: The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing HIF1A gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a HIF1A mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a HIF1A gene in a cell or in a subject to treat a HIF1A-related disease.
Type:
Application
Filed:
February 28, 2008
Publication date:
January 21, 2010
Applicant:
MDRNA, INC.
Inventors:
Steven C. Quay, James McSwiggen, Narendra K. Vaish, Mohammad Ahmadian
Abstract: The present disclosure provides a ribonucleic acid comprising a double-stranded region having at least one base pair comprising a 5-methyluridine base paired with a 2,6-diaminopurine and methods for preparing the same. Also provided are methods for treating or preventing a disease or disorder by inducing RNAi.
Abstract: Polynucleotide delivery-enhancing polypeptides are admixed or complexed with, or conjugated to, nucleic acids for enhancing delivery the nucleic acids into cells. The transported nucleic acids are active in target cells as small inhibitory nucleic acids (siNAs) that modulate expression of target genes, mediated at least in part by RNA interference (RNAi). The siNA/polypeptide compositions and methods of the invention provide effective tools to modulate gene expression and alter phenotype in mammalian cells, including by altering phenotype in a manner that eliminates disease symptoms or alters disease potential in targeted cells or subject individuals to which the siNA/polypeptide compositions are administered.
Type:
Application
Filed:
September 8, 2008
Publication date:
February 12, 2009
Applicant:
MDRNA, INC.
Inventors:
Michael E. Houston, JR., Lishan Chen, Kunyuan Cui, Yuching Chen, Sasha J. Mayer
Abstract: A double-stranded RNA (dsRNA) molecule comprising between about 15 base pairs and about 40 base pairs, wherein at least one ribonucleotide of the dsRNA is a 5?-methyl-pyrimidine and/or at least one 2?-O-methyl ribonucleotide, and a method of improving ribonuclease stability, reducing off-target effects of a double stranded siRNA molecule, or of reducing interferon responsiveness of a double stranded siRNA molecule using such dsRNA.
Type:
Application
Filed:
May 25, 2006
Publication date:
January 15, 2009
Applicant:
MDRNA, INC
Inventors:
Steven C. Quay, Kunyuan Cui, Paul H. Johnson, Lishan Chen, Mohammad Ahmadian
Abstract: Pharmaceutical compositions and methods are described comprising at least a parathyroid hormone peptide (PTH) preferably PTH1-34 and one or more mucosal delivery-enhancing agents for enhanced nasal mucosal delivery of PTH, for treating or preventing osteoporosis or osteopenia in a mammalian subject, preferably a human.
Type:
Grant
Filed:
October 17, 2006
Date of Patent:
October 14, 2008
Assignee:
MDRNA, Inc.
Inventors:
Steven C. Quay, Henry R. Costantino, Mary S. Kleppe, Ching-Yuan Li