The present disclosure is directed to methods and compositions useful for inserting and expressing a heterologous (exogenous) gene within a genomic locus, such as a safe harbor site, of a host cell.

A lyophilized composition capable of encapsulating any nucleic acid with high efficiency and easily is provided. A lyophilized composition of lipid nanoparticles not containing a nucleic acid but containing an ionic lipid, a sterol, a PEG lipid, an acidic buffer component that shows a buffering action at pH 1-6, and a cryoprotectant, wherein a weight ratio of the cryoprotectant and a total lipid is 10:1-1000:1.

Disclosed are methods for delivering a therapeutic or diagnostic agent to the cytosol of a cell in a subject. The disclosed methods generally include administering to the subject an effective amount of a lipid nanoparticle comprising the therapeutic or diagnostic agent and an effective amount of a membrane-destabilizing polymer. Also disclosed are related compositions and delivery systems.

The present invention relates to an orally-administered gene carrier and a use thereof, and more specifically, to: an oral gene carrier comprising, at the C-terminus of an immunoglobulin Fc region, a linker formed from cationic arginine and enabling the condensation of an anionic gene; and an oral composition for preventing, ameliorating or treating metabolic diseases, the composition comprising the gene carrier and the GLP-1 gene as active ingredients. The gene carrier, according to the present invention, may be usefully employed as an orally-administered carrier for various genes, and especially, is expected to be usable for preventing, ameliorating or treating metabolic diseases, such as diabetes and obesity, by effectively transferring the GLP-1 gene.

Provided is a polydixylitol polymer gene transporter (VBXYP-P) containing vitamin B6 and a cancer stem cell-targeting peptide (TR-7) and a method for preparing the same. In addition, provided is a nucleic acid delivery complex in which a therapeutic nucleic acid is conjugated to the gene transporter, and a pharmaceutical composition for gene therapy containing the complex as an active ingredient. In addition, provided is the gene transporter, a gene delivery complex, and gene therapy using the same. It was observed that VBXYP-P of the present disclosure had a remarkably higher nucleic acid delivery rate to cancer stem cells than the pre-existing nucleic acid transporter, and when VBXYP-P was conjugated with DNA, the complex was almost free of cytotoxicity and permeated a blood brain barrier to exhibit remarkably high transformation efficiency for cancer stem cells inside a brain tumor.

The present invention provides DNA targeting systems and methods for delivery of gene editing machinery using lipid nanoparticles or microparticles.

Embodiments of the invention concern copolymers and nanoparticles for use as delivery agents for one or more agents for therapy for a medical condition of humans and animals. Some of embodiments of the invention provide new reagents for biomedical research in cell culture, animal models and plants, for example. The copolymers comprise PLGA and PEI and, in some embodiments, also comprise 1-(3-aminopropyl)-4-methylpiperazine (APMP), Fc binding peptide and/or antibody. In certain embodiments, APMP-PLGA-PEI, Fc binding peptide/antibody-PLGA-PEI or Fc binding peptide/antibody-APMP-PLGA-PEI nanoparticles comprising one or more therapeutic agents are delivered to an individual in need thereof or used for biomedical research in cell cultures, animal models and plants.

The disclosure pertains to a recombinant adeno-associated virus (rAAV) comprising an Anc80L65 capsid for delivering a polynucleotide (e.g., a transgene) into the central nervous system (CNS). Further provided includes methods for treating CNS diseases using the rAAV and pharmaceutical compositions comprising the rAAV.

Disclosed are cationic lipids which are compounds of Formula (A). Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

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Disclosed are the use of one or more lipid compounds in the delivery of nucleic acids, and a lipid-nucleic acid mixture, a pharmaceutical composition or a kit comprising the lipid compound and nucleic acids. The lipid compounds provided in the present invention can promote the absorption of nucleic acids, particularly via oral absorption, and can promote the entry of the nucleic acids at target sites in a subject in need thereof.