A method for reducing or substantially preventing soft tissue calcification is provided. The method includes administering at least one of a downregulator of at least one plasmin inhibitors and plasmin(ogen) to a subject in need thereof, wherein the at least one plasmin inhibitor includes alpha2-antiplasmin. Further disclosed is a method comprises administering the compound subsequent to muscle injury, and the method further comprising administering an antifibrinolytic.

The present invention mainly relates to a DNA structure for use in treating an ocular pathology and for the non-viral transfer of nucleic acids into the muscular cells of the eyeball of a patient suffering from the ocular pathology; characterised in that it particularly comprises a first sequence encoding a first therapeutic protein and a second sequence encoding a second therapeutic protein which is different from the first therapeutic protein, the DNA structure being administered to the patient by injection into a ciliary muscle then electrotransfer into the cells of the ciliary muscle.

Compositions useful for treatment of Spinal and Bulbar Muscular Atrophy (SBMA) comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding at least one hairpin forming miRNA that comprises a targeting sequence which binds a target site on the mRNA of human androgen receptor, wherein the miRNA inhibits expression of human androgen receptor, is provided. Also provided are compositions containing a rAAV vector and methods of treating SBMA in patient comprising administration of a rAAV vector.

A compound, a liposome, a method for preparing a liposome, a drug carrier, a pharmaceutical composition and a use of the pharmaceutical composition in the treatment or prevention of hyperlipidemia and related diseases thereof.

Disclosed are nanoparticles that are introduced into cells and express a specific protein and a manufacturing method thereof. More particularly, the present invention relates to mRNA nanoparticles, which increase the expression of a specific protein capable of stimulating the cellular immune system to induce cellular immune responses and are thus applicable to treat a variety of diseases, do not require passage across the nuclear envelope because a desired gene is delivered not as plasmid DNA itself but in the form of mRNA, thus improving the efficiency of protein expression, and the nanoparticles are generated through a one-step process with a relatively small amount of plasmid DNA via rolling circle transcription (RCT), thereby providing a simple and economical process for gene delivery. The present invention is also concerned with such mRNA nanoparticles.

In some aspects, the disclosure relates to methods and compositions for modulation of miR-122 expression in a cell or a subject. In some embodiments, methods and compositions described by the disclosure are useful for the treatment of liver-associated diseases (e.g., chronic liver disease, alcoholic liver disease).

Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions.

Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.

The present invention relates to methods of treating neuropathy patients who have been administered a gabapentinoid. In particular, the methods involve administering a nucleic acid construct encoding human HGF proteins after discontinuing gabapentinoid. The present invention provides a novel method for a specific patient population to achieve a better therapeutic outcome by avoiding interference of therapeutic effects by gabapentinoids.