Abstract: Methods and materials are described for producing immortalized hybrid cells composed of a fusion of immortalized multi-lineage progenitor cells (MLPC) and primary hepatocytes. The hybrid cells express the biological activity of the primary hepatocytes and the immortality and expansion capacities of the immortalized MLPC. The methods of culture and expansion of the resultant hybrid cells and the methods to confirm the characteristics of the hybrid cells are described.

Abstract: This disclosure describes liver-specific nanocapsules for specifically targeting liver cells. This disclosure also provides methods of using such liver-specific nanocapsules to deliver one or more cargo moieties to the liver cells.

Abstract: This disclosure describes liver-specific nanocapsules for specifically targeting liver cells. This disclosure also provides methods of using such liver-specific nanocapsules to deliver one or more cargo moieties to the liver cells.

Abstract: The present invention provides methods for treating visual disorders. Exemplary visual disorders include macular degeneration, retinitis pigmentosa, glaucoma, and/or retinal degeneration.

Abstract: A method is disclosed for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance to the infusion site of the brain, the at least one substance capable of altering a nucleotide in a DNA sequence of a gene to convert a codon in a protein-coding region of the gene into a stop codon in the brain, whereby neurodegeneration in the brain is reduced. In a preferred embodiment, the at least one substance is a mutational vector, for example, a RNA/DNA chimeric mutational vector. The disclosed invention provides a method for treating neurodegenerative disorders such as Huntington's disease, spinocerebellar ataxia type 1, type 2, type 3, type 6, and/or type 7, spinobulbar muscular atrophy (Kennedy's disease), and/or dentatorubral-pallidoluysian atrophy (DRPLA).

Abstract: Methods for limiting apoptosis in a cell population by contacting such cells with a hydrophilic bile acid, such as ursodeoxycholic acid (UDCA), salts thereof, and analogs thereof (e.g., glyco- and tauro-ursodeoxycholic acid).

Abstract: Methods for limiting apoptosis in a cell population by contacting such cells with a hydrophilic bile acid, such as ursodeoxycholic acid (UDCA), salts thereof, and analogs thereof (e.g., glyco- and tauro-ursodeoxycholic acid).

Abstract: The present invention concerns compositions and methods for the introduction of specific genetic changes in endogenous genes of the cells of an animal. The genetic changes are effected by oligonucleotides or oligonucleotide derivatives and analogs, which are generally less than about 100 nucleotides in length. The invention provides for macromolecular carriers, optionally incorporating ligands for clathrin coated pit receptors. In one embodiment the ligand is a lactose or galactose and the genetic changes are made in hepatocytes. By means of the invention up to 40% of the copies of a target gene have been changed in vitro. Repair of mutant genes having a Crigler-Najjar like phenotype and Hemophilia B phenotype were observed.

Abstract: The present invention provides cationic polymers that include a primary amine and a targeting group covalently bound to the primary amine, wherein the targeting group targets a cell of interest by interacting with the surface of the cell. The invention also provides molecular complexes that include a polyethyleneimine and a targeting group covalently bound to a primary amine of the polyethyleneimine, and a biologically active compound. The invention further provides methods for delivering a biologically active compound to a vertebrate cell.