A peptide can have a sequence of one of SEQ ID NOs: 78-91. A conformationally-constrained kinked peptide includes: a conformationally-constraining portion and a kinked portion linked to the conformationally-constraining portion that conformationally constrains the kinked portion having a peptide sequence of one of SEQ NOs: 78-97. A cell-targeting compound can include a conformationally-constrained kinked peptide having a peptide sequence of one of SEQ ID NOs: 78-97. The peptide sequence can be one of SEQ ID NOs: 78-97, or 78-91, or 92-97. A cell-targeting compound can include a conformationally-constrained kinked peptide linked to a branched linker with one branch arm linked to a specific targeting moiety and one branch ann linked to a general targeting moiety. The specific targeting moiety can be an antibody. The general targeting moiety can be a lipid or cholesterol derivative.

The present invention provides targeting lipids of structure
L.sup.100-linker-L.sup.101  (CI),
where L.sup.100 is a lipid, lipophile, alkyl, alkenyl or alkynyl, L.sup.101 is a ligand or —CH.sub.2CH.sub.2(OCH.sub.2CH.sub.2).sub.pO(CH.sub.2).sub.qCH.sub.2-ligand, p is 1-1000, and q is 1-20. In addition, the invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo.

Aspects of the present invention include the production and use of chemically modified RNAi agents (e.g., shRNAs) in gene silencing applications. The chemically modified RNAi agents disclosed herein have reduced immunostimulatory activity, increased serum stability, or both, as compared to a corresponding RNAi agent not having the chemical modification. Compositions containing chemically modified RNAi agents according to aspects of the present invention (including pharmaceutical compositions) and kits containing the same are also provided.

The invention relates to compounds, compositions and polymers comprising a first component adapted to promote germination of Clostridium difficile (C.diff) and a second component which acts as an antimicrobial agent. Said compounds, compositions and polymers are useful for destroying C.diff where conventional antimicrobial agents are unsuccessful. The compositions can be formulated as coating or materials which actively destroy C.diff which come into contact with it. The germination promotion is induced by bile salts. The invention also relates to the use of such materials as a treatment for C.diff associated diseases and toxic megacolon.

Described herein are doppel-targeting molecules useful for inhibiting pathological angiogenesis and treating diseases and conditions associated with pathological angiogenesis, such as tumors, cancers, atherosclerosis, tuberculosis, asthma, pulmonary arterial hypertension (PAH), neoplasms and neoplasm-related conditions, and for detecting doppel expression in a subject. Related compositions and methods also are described.

The present invention relates to nanoparticles complexed with biomacromolecule agents configured for treating, preventing or ameliorating various types of disorders, and methods of synthesizing the same. In particular, the present invention is directed to compositions comprising nanoparticles (e.g., synthetic high density lipoprotein (sHDL)) carrying biomacromolecule agents (e.g., nucleic acid, peptides, glycolipids, etc.), methods for synthesizing such nanoparticles, as well as systems and methods utilizing such nanoparticles (e.g., in diagnostic and/or therapeutic settings).

The invention relates to methods, compounds, compositions and vehicles for delivering 3-amino-1-propanesulfonic acid (3APS) in a subject, preferably a human subject. The invention encompasses compound that will yield or generate 3APS, either in vitro or in vivo. Preferred compounds include amino acid prodrugs of 3APS for use, including but not limited to the prevention and treatment of Alzheimer's disease

This disclosure relates to hyaluronic acid nanoparticles containing an anticancer agent such as a NADPH oxidase (NOX) inhibitor for targeted delivery to cancerous cells or tumors. In certain embodiments, the nanoparticles are made up of hyaluronic acid conjugated to hydrophobic moieties. In certain embodiments, the hydrophobic moieties are steroid based compounds, such as 5beta-cholanic acid.

Formulated and/or co-formulated liposomes comprising TLR prodrugs and/or TLR Lipid Moieties and methods of making the liposomes are disclosed herein. The TLR prodrug compositions comprise a drug moiety, a lipid moiety, and linkage unit that inhibit Toll-Like Receptor (e.g., TLR1/2, TLR4, and/or TLR7). The TLR prodrugs can be formulated and/or co-formulated into a liposome to provide a method of treating cancer, immunological disorders, and other disease by utilizing a targeted drug delivery vehicle.

A nucleic acid complex that exhibits an excellent antisense effect in the skeletal muscle and/or heart muscle, and a composition for treating or preventing a muscle disease that develops in the skeletal muscle, heart muscle, and the like having the nucleic acid complex as an active ingredient is disclosed. Also provided is a double-stranded nucleic acid complex in which a first nucleic acid strand that hybridizes to the transcription product of a target gene and has an antisense effect on the transcription product is annealed with a second nucleic acid strand that has a base sequence complementary to the first nucleic acid strand and is bound to cholesterol or analog thereof.