A method of reducing the level of a transcription product in a cell comprising contacting with the cell a composition comprising a double-stranded nucleic acid complex comprising a first nucleic acid strand annealed to a second nucleic acid strand, wherein: (i) the first nucleic acid strand hybridizes to the transcription product and comprises (a) a region consisting of at least 4 consecutive nucleotides that are recognized by RNase H when the strand is hybridized to the transcription product, (b) one or more nucleotide analogs located on 5 terminal side of the region, (c) one or more nucleotide analogs located on 3 terminal side of the region and (d) a total number of nucleotides and nucleotide analogs ranging from 8 to 35 nucleotides and (ii) the second nucleic acid strand comprises (a) nucleotides and optionally nucleotide analogs and (b) at least 4 consecutive RNA nucleotides.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.

The invention relates to aptamers which specifically bind to immunoglobulin G and their use in the purification of said protein.

One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.

What is described is a method for treating a fibrotic disease by administering a pharmaceutical composition comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications.

The present invention relates to an improved RNA transcription vector, which is very suitable for the production of mRNA for in vivo therapeutic purposes. The improvements in the vector reside in the presence of a translation enhancer (TE) and a nuclear retention element (NRS), especially when the latter is the Expression and Nuclear Retention Element (ENE) of Kaposi's sarcoma associated Herpes virus (KSHV).

This disclosure provides compositions and methods for delivery of a polynucleotide to an organism. More specific ally, this disclosure relates to compositions including a mixture of a polynucleotide and a cationic polysaccharide, and methods of providing such compositions to an organism, such as a pest (e.g., an insect, a nematode, a mollusk).

The present invention in general relates to an improved RNA transcription vector, which is very suitable for the production of mRNA for in vivo therapeutic purposes. The improvements in the vector in particular reside in the presence of a transcription enhancer and a nuclear retention element.

Recombinant adenovirus genomes that include a heterologous open reading frame (ORF) and a self-cleaving peptide coding sequence are described. The recombinant adenovirus genomes and recombinant adenoviruses produced by the disclosed genomes can be used, for example, in high-throughput assays to measure virus replication kinetics. Methods for measuring replication kinetics of a recombinant adenovirus are also described.