The present disclosure provides a drug carrier, a brain-targeting nanodrug based on CRISPR gene editing technology and a preparation method and use thereof. The nanodrug contains nanoparticles prepared by coupling Cas9/sgRNA and drug carriers. The drug carrier includes a polymer mPEG-P (GPMA, FPMA) and a polymer Ang-PEG-PGPMA, wherein a structural formula of the mPEG-P (GPMA, FPMA) is:

##STR00001##

a structural formula of the polymer Ang-PEG-PGPMA is:

##STR00002##

where n is 35-45, x1 is 15-20, y is 2-4, m is 75-85, and x2=x1. The guanidino group of the drug carrier can be combined with the ribonucleoprotein complex by electrostatic action, salt bridge formation, or hydrogen bonding action. Also provided are methods of suppressing and treating tumors at a gene level using the drug carrier to transport the therapeutic drug to the lesion site.

A modified nanoparticle for use in a therapeutic method, wherein the therapeutic method comprises the administration of the modified nanoparticle to an organism, the targeting of the modified nanoparticles to a specific site in the organism followed by an uptake of the modified nanoparticle into a cell, and wherein the modified nanoparticle is obtainable by a process comprising the steps of i) providing a nanoparticle and ii) contacting the nanoparticle with one or more antibodies as at a pH value of less than 7.0 so as to non-covalently bind the one or more antibodies via its/their Fc region onto the surface of the nanoparticle, wherein the nanoparticle provided in step i) is made of a material having at least one protonable or deprotonable group on the surface thereof and/or the one or more targeting moieties contacted with the nanoparticle in step ii) has at least one protonable or deprotonable group.

The field of the disclosure relates generally to cancer cell destruction and, more specifically, to cancer cell destruction by photo-magnetic irradiation mediated multimodal therapy using smart nanostructures.

The present disclosure relates to elastomeric lyotropic liquid crystal (E-LLC) and mesoporous elastomers, wherein the mesoporous elastomer possesses a combination of covalent chemical crosslinks and physical chain entanglement crosslinks. The production and use such mesoporous elastomers is also provided.

Provided are targeted structure-specific particulate-based delivery systems comprising: a nanoparticle; a PEG polymer coating on the surface of the nanoparticle; a targeting moiety conjugated on a surface of the nanoparticle and configured to promote specific binding to a cell surface molecule expressed by a target cell; and a biologically active agent in or on the nanoparticle, wherein the biologically active agent is selected to enhance a desired response in a target cell intracellularly or extracellularly. Methods of treating a disease or disorder administering the delivery system are contemplated.

Metastatic triple negative breast cancer (TNBC) still carries a dismal prognosis with the current treatment paradigms. The effectiveness of drug treatment for many solid tumors such as TNBC is limited by tumor heterogeneity, lack of tumor specificity, off-target toxicities, and transient therapeutic action(s). Strategies that provide tumor-specific, sustained concentrations of drugs to the tumors and tumor receptor-specific binding, while reducing off-target effects are needed to ensure sufficient tumor cell uptake within the primary and metastatic tumor microenvironment. The decreased non-specific adhesivity, receptor-targeted nanoparticle formulations (“DART” nanoparticles) of the invention were assessed for clinical potential in directing biological agents to the cell surface receptor Fn14, which is expressed in many solid cancer types, including TNBC primary tumors and metastatic lesions. They are contemplated for use against solid tumors, particularly brain tumors such as glioblastoma and breast cancer, including metastatic breast cancer.

An aqueous liquid suspension containing a coated drug—ion exchange resin complex comprising a core composed of an amphetamine complexed with a pharmaceutically acceptable ion-exchange resin and an uncoated amphetamine—ion exchange resin complex is provided. The coated amphetamine—ion exchange resin complex is in admixture with a polymer to form a matrix. Methods of making the coated complex and the liquid suspension are described.

The invention provides siRNA compositions that specifically downregulates expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD).

The present invention relates to the field of medicine, particularly to functionalised nanoparticles (NP) for use in cancer therapy (treatment or diagnosis), to pharmaceutical compositions or nano devices comprising same, and also to a method for obtaining said functionalised nanoparticles. The nanoparticles described in the present invention can be used specifically to treat cancer efficiently, as same can selectively detect tumour cells.

Provided herein are copolymers comprising a plurality of a first monomer and a plurality of a second monomer, wherein chloroquine, hydroxychloroquine, or a chloroquine analog is appended to at least a portion of the plurality of the first monomer. Also provided are nanoparticles comprising copolymers as described herein, and methods of using the copolymers and nanoparticles for treating diseases or disorders, e.g., Inflammatory Bowel Disease (IBD) or cancer (e.g., colon cancer).