The present invention provides compositions, systems, kits, and methods for expression of one or more biomolecules in a subject, human or non-human mammal, (e.g., at therapeutic levels for the extended periods of time required to produce therapeutic effects). In certain embodiments, compositions, systems, kits, and methods are provided that comprise a first composition comprising polycationic structures (e.g., empty cationic liposomes, cationic micelles, cationic emulsions, or cationic polymers) and a second composition comprising expression vectors (e.g., non-viral expression vectors not associated with liposomes or other carriers) encoding one or more biomolecules of interest.

The present invention provides compositions, systems, kits, and methods for expression of one or more biomolecules in a subject, human or non-human mammal, (e.g., at therapeutic levels for the extended periods of time required to produce therapeutic effects). In certain embodiments, compositions, systems, kits, and methods are provided that comprise a first composition comprising polycationic structures (e.g., empty cationic liposomes, cationic micelles, cationic emulsions, or cationic polymers) and a second composition comprising expression vectors (e.g., non-viral expression vectors not associated with liposomes or other carriers) encoding one or more biomolecules of interest.

An object of the present invention is to provide an animal cell with improved proliferation ability and survival rate, a method for producing the animal cell, and a method for producing a target protein formed of the animal cell. According to the present invention, there is provided an animal cell having a gene encoding a target protein and a foreign gene encoding an isovaleryl-CoA dehydrogenase, in which the isovaleryl-CoA dehydrogenase is overexpressed.

An object of the present invention is to provide an animal cell with improved proliferation ability and survival rate, a method for producing the animal cell, and a method for producing a target protein formed of the animal cell. According to the present invention, there is provided an animal cell having a gene encoding a target protein and a foreign gene encoding an isovaleryl-CoA dehydrogenase, in which the isovaleryl-CoA dehydrogenase is overexpressed.

The present invention provides a recombinant adipose-derived stem cell and a recombinant method thereof, and belongs to the technical field of genetic engineering, where an adenovirus carrying an hFIX gene is transfected into an adipose-derived stem cell to obtain the recombinant adipose-derived stem cell. In the present invention, an adenovirus carrying an hFIX gene is transfected into an adipose-derived stem cell, and the recombinant adipose-derived stem cell obtained after the transfection can express an hFIX protein.

The present invention provides a recombinant adipose-derived stem cell and a recombinant method thereof, and belongs to the technical field of genetic engineering, where an adenovirus carrying an hFIX gene is transfected into an adipose-derived stem cell to obtain the recombinant adipose-derived stem cell. In the present invention, an adenovirus carrying an hFIX gene is transfected into an adipose-derived stem cell, and the recombinant adipose-derived stem cell obtained after the transfection can express an hFIX protein.

The present invention concerns platelets transfected with exogenous genetic material and microparticles deriving from said transfected platelets having a high percentage of transfection and able to transport and to transfect acceptor cells with genetic material and then used for example in gene and cell therapy. The invention further concerns a method for the preparation of mature platelets transfected with exogenous genetic material and microparticles deriving from said transfected platelets and microparticles deriving from said transfected mature platelets which permits to obtain high percentages of transfection.

The present invention concerns platelets transfected with exogenous genetic material and microparticles deriving from said transfected platelets having a high percentage of transfection and able to transport and to transfect acceptor cells with genetic material and then used for example in gene and cell therapy. The invention further concerns a method for the preparation of mature platelets transfected with exogenous genetic material and microparticles deriving from said transfected platelets and microparticles deriving from said transfected mature platelets which permits to obtain high percentages of transfection.

The present disclosure provides, in part, nucleic acid loaded flowable hydrogels and compositions, systems and methods related thereto, to effectively deliver nucleic acids to cells that contact the flowable hydrogels.

Hyperbranched cationic polymers are described. The polymers employ a 4-branching monomer resulting in an increase in the number of functional terminal groups due to the extra branching units, providing excellent transfection efficiency and cytocompatibility in different cell types, including aADSC, HeLa, Neu7 and RDEB keratinocytes, and delivering different genetic therapy approaches such GFP plasmid DNA and a ribonucleoprotein CRISP-Cas 9 complex for COL7A1 exon 80 skipping. In addition, the extra branching units of the polymer of the invention increases the positive charge on the polymer, which provides for improved endosomal escape within the cell. The 4-branching unit can be a diamine component, or a tetraacrylate component, although other 4-branching monomers may be employed such as for example any component with tetra acrylamide groups (i.e. 4-arm PEG acrylamide, 4-arm PEG maleimide), any component with tetra N- hydroxysuccinimide (NHS) groups (i.e. 4-arm PEG-succinimidyl carbonate NHS ester), any type of tetrathiol component (i.e. Pentaerythritol tetrakis(3-mercaptopropionate), 4-arm PEG-thiol, Tetra(2- mercaptoethyl)silane), and any tetraepoxy component (i.e. TetraGlycidyl methylenedianiline, Tetraglycidyl 1, 1′-methylenebis(naphthalene-2,7-diol), Pentaerythritol tetraglycidyl ether, 4-arm peg epoxide).