Abstract: The present disclosure includes cationic carrier units comprising (i) a water-soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.

Abstract: The present disclosure includes the use of miRNA inhibitor for treating a disease or condition associated with a decreased level of PSD95 and/or synaptophysin and/or an increased level of caspase 3 (e.g., dystonia, neuropsychiatric diseases, intellectual disability, and/or addiction). The miRNA inhibitor useful for the present disclosure can inhibit miR-485 expression and/or activity, which in turn can increase the level of PSD95 and/or synaptophysin protein of gene expression and decrease the level of caspase 3 protein or gene expression.

Abstract: The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moeity, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an RNA and/or DNA) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.

Abstract: The present disclosure includes the use of a miRNA inhibitor for inducing hair growth, increasing the hair density, increasing the follicular density, increasing the hair shaft thickness, increasing hair length, preventing hair loss, reducing hair loss, or any combination thereof in a subject in need thereof. In some aspects, the subject has one or more disorders selected from the group consisting of alopecia greata, androgenic alopecia, alopecia areata, alopecia universalis, involutional alopecia, trichotillomania, telogen effluvium, anagen effluvium, cicatricial, alopecia, scarring alopecia, scalp thinning, hair shaft abnormalities, infectious hair disorders, genetic disorders, and hair loss due to chemotherapy, hormonal imbalance, fungal infection, medication intake, chemical hair treatment, or aging.

Abstract: The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, and (iii) an adjuvant moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.

Abstract: The present disclosure includes the use of a miRNA inhibitor for treating a symptom or condition of Huntington’s disease. The miRNA inhibitor useful for the present disclosure can inhibit miR-485 expression and/or activity, which in turn can modulate the level of proteins or gene expression related to Huntington’s disease.

Abstract: The present invention relates to a composition for preparing an Alzheimer's disease animal model using microRNA, a non-human Alzheimer's disease animal model, and a method for screening compounds capable of treating Alzheimer's disease using the same.

Abstract: The present disclosure relates to the use of miR-485-3p expression to identify a subject that is afflicted with a cognitive disorder. In some aspects, the methods disclosed herein further comprises administering a miR-485-3p inhibitor to the subject, wherein the miR-485-3p inhibitor is capable of treating the cognitive disorder.

Abstract: The present disclosure includes the use of a miRNA inhibitor for treating amyotrophic lateral sclerosis (ALS) associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1? protein and/or PGC-1? gene expression, CD36 protein and/or CD36 gene expression, NRG1 protein and/or NRG1 gene expression, STMN2 protein and/or STMN2 gene expression, and/or NRXN1 protein and/or NRXN1 gene expression.

Abstract: The present disclosure includes the use of a miRNA inhibitor for treating a disease or condition associated with a decreased level of SIRT1, PGC-1?, CD36, LRRK2, NRG1, STMN2, VLDLR, NRXN1, GRIA4, NXPH1, PSD-95, and/or synaptophysin protein or SIRT1, PGC-1?, CD36, LRRK2, NRG1, STMN2, VLDLR, NRXN1, GRIA4, NXPH1, PSD-95, and/or synaptophysin gene expression. In some aspects, the miRNA inhibitor can be used to treat a disease or condition associated with an increased level of caspase-3 protein or gene expression. The miRNA inhibitor useful for the present disclosure can inhibit miR-485 expression and/or activity, which in turn can increase the level of SIRT1, PGC-1?, CD36, LRRK2, NRG1, STMN2, VLDLR, NRXN1, GRIA4, NXPH1, PSD-95, and/or synaptophysin protein or gene expression; and/or can decrease the level of caspase 3 protein or gene expression.

Abstract: The present disclosure relates to the use of PGC-1? expression to identify a subject that is conducive to treatment with a ma-485 inhibitor. In some aspects, the subject suffers from a disease or disorder associated with reduced PGC-1? expression. In some aspects, the PGC-1? expression is measured in the serum of the subject.

Abstract: The present disclosure relates to the use of SIRT1 expression to identify a subject that is conducive to treatment with a miR-485 inhibitor. In some aspects, the subject suffers from a disease or disorder associated with reduced SIRT1 expression. In some aspects, the SIRT1 expression is measured in the serum of the subject.

Abstract: The present disclosure includes the use of a miRNA inhibitor for treating a tauopathy associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1? protein and/or PGC-? gene expression, and/or CD36 and/or CD36 gene expression.

Abstract: The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, and (iii) an adjuvant moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.

Abstract: The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, and (iii) an adjuvant moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.

Abstract: The present invention provides a polyion complex micelle including an antisense oligonucleotide and a block copolymer of a cationic polymer and a PEG block having a number average molecular weight of 3 kDa to 10 kDa.

Abstract: A tumor-targeting gas-generating nanoparticle, a method for preparing same and a tumor-targeting nanoparticle for drug delivery using same relate to a tumor-targeting gas-generating nanoparticle including a polycarbonate core and a amphiphilic coat, a method for preparing same and a tumor-targeting nanoparticle for drug delivery using same. Since a tumor-targeting gas-generating nanoparticle according to the present disclosure is accumulated in the tumor tissue in large quantity and generates strong ultrasound wave signals, it can be usefully used as a contrast agent for ultrasonic imaging.

Abstract: Disclosed is a liver tumor-targeting ultrasound contrast agent. The ultrasound contrast agent includes a gas-generating core and a hyaluronic acid shell. The ultrasound contrast agent can be specifically delivered to liver cells. This specific delivery enables easy differentiation between normal liver cells and liver tumor cells by ultrasound imaging. In addition, the ultrasound contrast agent is highly stable in aqueous condition and causes no cytotoxicity. Also disclosed is a method for preparing the ultrasound contrast agent.

Abstract: Disclosed is a liver tumor-targeting ultrasound contrast agent. The ultrasound contrast agent includes a gas-generating core and a hyaluronic acid shell. The ultrasound contrast agent can be specifically delivered to liver cells. This specific delivery enables easy differentiation between normal liver cells and liver tumor cells by ultrasound imaging. In addition, the ultrasound contrast agent is highly stable in aqueous condition and causes no cytotoxicity. Also disclosed is a method for preparing the ultrasound contrast agent.

Abstract: A tumor-targeting gas-generating nanoparticle, a method for preparing same and a tumor-targeting nanoparticle for drug delivery using same relate to a tumor-targeting gas-generating nanoparticle including a polycarbonate core and a amphiphilic coat, a method for preparing same and a tumor-targeting nanoparticle for drug delivery using same. Since a tumor-targeting gas-generating nanoparticle according to the present disclosure is accumulated in the tumor tissue in large quantity and generates strong ultrasound wave signals, it can be usefully used as a contrast agent for ultrasonic imaging.