The present invention relates to a microbubble complex comprising a microbubble having an outer shell comprising a mixture of native and denatured albumin encapsulating a perfluorocarbon gas, a therapeutic agent, a bifunctional linker having one end attached to the therapeutic agent and the other attached to a ligand and wherein the ligand is bound to the other shell of the microbubble through hydrophobic interactions. Also included are methods for delivering the aforementioned microbubble complex to a tissue target.

The invention discloses the use of single-stranded RNA toeholds of different lengths to promote the re-association of various RNA-DNA hybrids, which results in activation of multiple split functionalities inside human cells. Previously designed RNA/DNA nanoparticles employed single-stranded DNA toeholds to initiate re-association. The use of RNA toeholds is advantageous because of the simpler design rules, the shorter toeholds, and the smaller size of the resulting nanoparticles compared to the same hybrid nanoparticles with single-stranded DNA toeholds. Moreover, the co-transcriptional assemblies result in higher yields for hybrid nanoparticles with ssRNA toeholds.

Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.

The present invention relates to compositions comprising transmembrane stem cell factor (tmSCF) polypeptide embedded in a lipid vesicle and methods of use thereof. In some embodiments, the lipid vesicle is a nanocarrier. Disclosed herein is a method for promoting angiogenesis in a subject, comprising administering to a subject in need thereof an effective amount of a composition comprising a tmSCF polypeptide embedded in a lipid vesicle. Disclosed herein is a method for treating a subject with peripheral vascular disease (PVD), comprising administering to a subject in need thereof a therapeutically effective amount of a composition comprising a tmSCF polypeptide.

Compositions and methods for treating cancer in a subject in need thereof are described that includes administering a therapeutically effective amount of an oligonucleotide that specifically hybridizes to MALAT1.

The present invention pertains to a complex including nanoparticles and, carried on the surface of the nanoparticles, a lysosomal enzyme inhibitor or kinase inhibitor shown by general formula (A) and a phospholipid mimetic substance shown by general formula (B). ##STR00001## In general formula (A), n1 is an integer of 2-30, n2 is an integer of 2-30, the -S- terminal is a nanoparticle-carrying site, and R10 is a suicide substrate site or a kinase inhibition site. In general formula (B), n3 is an integer in the range of 2-30, and the -S- terminal is a nanoparticle-carrying site. The present invention provides a versatile system capable of efficiently delivering a drug to endolysosomes and allowing the drug to function at a low concentration on lysosomal enzymes, and an anticancer agent in which this system is used.

A bone repair material, a preparation method of the bone repair material, and a biological composite scaffold are provided. The bone repair material includes: a base material, and a carbon nanomaterial and a polypeptide both mixed with the base material; and the carbon nanomaterial and the polypeptide are bonded by chemical bonds. The preparation method includes: bonding a carbon nanomaterial with a polypeptide by chemical bonds; and mixing the carbon nanomaterial and the polypeptide bonded by the chemical bonds with a base material, and performing a molding treatment.

The present disclosure provides phospholipid-containing compounds, pharmaceutical compositions and microspheres that exhibit high affinity for mineralized metals. The present disclosure also provides strategies for using said compounds, compositions and microspheres in the treatment of nephrolithiasis or kidney stone disease, and methods of manufacturing and preparing said compounds and compositions.

A method of delivering drugs to inner ear facilitated by microbubbles, including mixing a microbubble composition and a drug into a microbubble-drug mixture, applying the microbubble-drug mixture to middle ear cavity, and placing a mechanical oscillation wave source to ear canal or cranium located behind the ear. The mechanical waves generated by the mechanical oscillation wave source penetrate through tympanum or cranium, and induce the cavitation on the microbubbles in the middle ear cavity. Thus, the permeability of the round window membrane is increased, so that the drug penetrates into inner through the round window membrane. Therefore, the mechanical oscillation wave source induces the cavitation on the microbubbles in a non-invasive way.

The present subject matter relates to techniques for treating a neurodegenerative disease. The disclosed system can include a transducer for stimulating a target tissue with focused ultrasound (FUS) and at least one nanocup. The transducer induces the FUS with a predetermined parameter to open the target tissue. The nanocup can include at least one gas pocket within a cavity of the nanocup and an effective amount of an active agent for neuroregeneration.