C12N2760/16043

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

Methods are provided for the cell-based delivery of collagen VII for the treatment of Epidermolysis Bullosa and corneal erosion. The disclosure also provides a composition and a pharmaceutical composition comprises, comprise, or alternatively consist essentially of, or yet further consist of a keratinocyte sheet or a corneal cell sheet.

RECOMBINANT VECTORS

This disclosure provides modified cytosine deaminases (CDs). The disclosure further relates to cells and vector expressing or comprising such modified CDs and methods of using such modified CDs in the treatment of disease and disorders.

PLASMIDS AND METHOD FOR OBTAINING VIRAL PARTICLES

The present invention describes a viral RNA expression plasmid and a method for obtaining viral particles based on said plasmids comprising transfecting animal cells with an expression vector or a set of expression vectors capable of expressing a nucleoprotein and RNA-dependent polymerase RNA; and transfecting the animal cell with an expression vector or a set of expression vectors with nucleotide sequences encoding recombinant RNA molecules.

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

Methods are provided for the cell-based delivery of collagen VII for the treatment of Epidermolysis Bullosa and corneal erosion. The disclosure also provides a composition and a pharmaceutical composition comprises, comprise, or alternatively consist essentially of, or yet further consist of a keratinocyte sheet or a corneal cell sheet.

GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING GENETICALLY CORRECTED AUTOLOGOUS KERATINOCYTES

Methods are provided for the cell-based delivery of collagen VII for the treatment of Epidermolysis Bullosa and corneal erosion. The disclosure also provides a composition and a pharmaceutical composition comprises, comprise, or alternatively consist essentially of, or yet further consist of a keratinocyte sheet or a corneal cell sheet.

GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING GENETICALLY CORRECTED AUTOLOGOUS KERATINOCYTES

Methods are provided for the cell-based delivery of collagen VII for the treatment of Epidermolysis Bullosa and corneal erosion. The disclosure also provides a composition and a pharmaceutical composition comprises, comprise, or alternatively consist essentially of, or yet further consist of a keratinocyte sheet or a corneal cell sheet.

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

Methods are provided for the cell-based delivery of collagen VII for the treatment of Epidermolysis Bullosa and corneal erosion. The disclosure also provides a composition and a pharmaceutical composition comprises, comprise, or alternatively consist essentially of, or yet further consist of a keratinocyte sheet or a corneal cell sheet.

NEDDYLATION-ACTIVATING ENZYME INHIBITORS AS VIRAL SENSITIZERS AND USES THEREOF

The present application relates to viral sensitizers. More specifically, the present application relates to neddylation-activating enzyme inhibitors, as well as processes for their preparation and methods of using such compounds and compositions as viral sensitizers. The present application includes a method of increasing permissiveness of a cell to a virus or genetic material encoding components of the virus, comprising administering an effective amount of a neddylation-activating enzyme (NAE) inhibitor, or a salt, solvate and/or prodrug thereof, to the cell.

Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

Methods are provided for the cell-based delivery of collagen VII for the treatment of Epidermolysis Bullosa and corneal erosion. The disclosure also provides a composition and a pharmaceutical composition comprises, comprise, or alternatively consist essentially of, or yet further consist of a keratinocyte sheet or a corneal cell sheet.