Abstract: The invention provides siRNA compositions that interfere with viral replication in respiratory viral infections, including respiratory syncytial virus and avian influenza A, including the H5N1 strain. The invention further provides uses of the siRNA compositions to inhibit expression of viral genes in respiratory virus-infected cells, and to uses in the treatment of respiratory virus infections in a subject. Generally the invention provides polynucleotide that includes a first nucleotide sequence of 15 to 30 bases that targets the genome of a respiratory syncytial virus or an influenza A virus, a complement thereof, a double stranded polynucleotide or a hairpin polynucleotide. Additionally the invention provides vectors, cells and pharmaceutical compositions containing siRNA sequences.

Abstract: Biomedical nanoparticles are disclosed based on new engineered modular carrier macromolecules, on engineered macromolecules or associated entities providing an internal nanoparticle structure, and compositions for minimizing non-specific binding of the nanoparticles while enabling efficient and convenient targeting to cells and tissues. These nanoparticles may be used to deliver atomic or molecular or associated entities which are useful for diagnostics, primarily in vivo imaging, for therapeutics, for vaccines, or for experimental research. Nanoparticles comprising combinations of active entities such as gene inhibitors with gene expression cassettes or imaging agents with therapeutic agents, and polyamide compounds useful for treatment of microbial infections are also disclosed.

Abstract: This invention describes compositions and methods using siRNA to target various genes expressed in cells of transplanted organs or tissues and/or genes expressed in the host to improve the success of the transplantation.

Abstract: The present invention embodies a multi-disciplinary approach to validate or identify targets involved in any given biological process or pathway, such as an immune response, or progression or regression of disease. By introducing a target(s) with, e.g., gene delivery vector(s) or other foreign substance(s) to an in vivo system and by integrating, for example, pathological, pharmacological, bioassay, microarray and bioinformatics data obtained from the in vivo system, the present inventors are able to (1) identify one or more targets, e.g., genes, that are involved in a biological pathway of interest, (2) implement these identified targets for further analysis of the biological process or pathway and (3) provide a scalable approach in vivo for potential large quantities of target(s) discovery and validation. This process can be used in any number of applications, including the identification of agonists and antagonists to a biological process or pathway, which can lead to drugs and vaccine discovery.

Abstract: It has been found that certain synthetic vectors and nucleic acid sequences that encode viral genomic sequences can, for example, be administered to a subject repeatedly as a vehicle for effectively delivering one or more therapeutic nucleic acid molecules or polypeptides to a cell or tissue. Accordingly, the disclosed nucleic acid delivery vehicles can be used, for instance, as part of a therapeutic regimen that involves an ongoing use of a therapeutic nucleic acid molecule or polypeptide.

Abstract: The present invention provides compositions and methods that are useful for the treatment of severe acute respiratory syndrome (SARS). More specifically, nucleic acid agents such as siRNA molecules and their analogues that target respiratory infections including SARS coronavirus and their methods of use are described, for clinical treatments of SARS, respiratory viral infections, for prevention and treatment of respiratory infections as needed for bio-defense, for treatment of respiratory diseases, and for discovery of therapeutic targets for respiratory diseases and infections.

Abstract: A liposome composition comprising small, surface-bound effector molecules is disclosed. The liposomes have a surface layer of hydrophilic polymer chains, for enhanced circulation time in the bloodstream. The effector molecules are attached to the distal ends of the polymer chains. In one embodiment, the effector is polymyxin B, for treatment of septic shock.

Abstract: The present invention relates to methods for treating cancers by manipulating a target gene expression by up-regulation, silencing and/or down-regulation of the gene, such as EGFR-RP, TRA1, MFGE8, TNFSF13 and ZFP236, respectively. The methods are useful in treating cancers and/or inhibiting tumor growth by enhancing expression of a gene that is validated as a target such as ICT1030, for protein, peptide drug and gene therapy modalities; or by RNA interference to silence and/or down-regulate targets such as ICT1024, ICT1025 and ICT1031 and ICB1003 that are validated for antibody, small molecule and other inhibitor drug modalities.

Abstract: Methods and compositions are provided for down regulation of target gene expression in vivo by RNA interference. The methods are useful for target discovery and validation of gene-based drug development, and for treatment of human diseases.

Abstract: Certain hydrophilic polymers, such as a polyoxazoline, when conjugated to a polypeptide or small molecule agent, can enhance the bioavailability of the agent when administered in vivo. Accordingly, hydrophilic polymers of the invention can be used as a delivery vehicle to treat any number of disorders and/or confer a myriad of therapeutic benefits to a subject.

Abstract: The inventions provide compositions and methods for nucleic acid delivery comprising IIPMA conjugated to a polyamine. These compositions have the benefit of the steric hindrance of HPMA and the nucleic acid binding capability of a polyamine. Useful polyamines for this purpose include spermine, spermidine and their analogues, and DFMO. These polyamines have the ability not only to bind nucleic acids, but also have anti-cancer effects themselves. The compounds provided can also include ligand binding domains, such as vascular endothelial growth factors, somatostatin and somatostatin analogs, transferring, melanotropin, ApoE and ApoE peptides, von Willebrand's factor and von Willebrand's factor peptides, adenoviral fiber protein and adenoviral fiber protein peptides, PD 1 and PD 1 peptides, EGF and EGF peptides, RGD peptides, CCK peptides, antibody and antibody fragments, folate, pyridoxyl and sialyl-LewisX and chemical analogs.

Abstract: A suspension of heterogeneous size liposomes is passed through a polymer filter having a web-like construction providing a network of interconnected, tortuous-path capillary pores, and a membrane thickness of at least about 100 microns. The processed liposomes have a selected average size less than about 0.4 microns, and a narrow size distribution.