Abstract: The invention provides methods for treating ocular diseases using a recombinant vehicle to express a protein useful in the treatment of ocular disease, with particular preference for use of neurotrophin-4 (NT4) for targeting subpopulations of cells in the retina. A genetically engineered gene transfer vector containing sequences encoding a growth factor such as neurotrophin-4 (NT4) is used to transduce cells of the retinal ganglion cell (RGC) layer, in situ, via administration of the vector intravitreally. Accordingly, methods are disclosed for treating subjects in need thereof by therapeutic protein delivery via a recombinant expression vector, including rescue of photoreceptors by targeting the RGC layer subpopulation of retinal cells.

Abstract: The invention provides methods for treating ocular diseases using a recombinant vehicle to express a protein useful in the treatment of ocular disease, with particular preference for use of neurotrophin-4 (NT4) for targeting subpopulations of cells in the retina. A genetically engineered gene transfer vector containing sequences encoding a growth factor such as neurotrophin-4 (NT4) is used to transduce cells of the retinal ganglion cell (RGC) layer, in situ, via administration of the vector intravitreally. Accordingly, methods are disclosed for treating subjects in need thereof by therapeutic protein delivery via a recombinant expression vector, including rescue of photoreceptors by targeting the RGC layer subpopulation of retinal cells.

Abstract: A specific clinical protocol for use toward therapy of defective, diseased and damaged neurons in the mammalian brain, of particular usefulness for treatment of neurodegenerative conditions such as Parkinson's disease. The protocol is practiced by directly delivering a definite concentration of a nerve growth factor via delivery of the protein, an expression vector operably encoding the nerve growth factor, or grafting a donor cell containing such an expression vector into the substantia nigra and preferably also the striatum. The method stimulates growth of targeted neurons, and reversal of functional deficits associated with the neurodegenerative disease being treated.

Abstract: The needle assembly of the invention is a quad-partite system for use with an arc-center stereotactic headframe that allows a clinician to deliver recombinant expression vectors through the needle assembly to targeted sites at exact depths in the brain. Methods for use of the needle assembly with a rube insert for the needle cannula that does not bind proteins, as in protein viral capsids, permit delivery of precise volumes of pharmaceutical compositions containing viral recombinant expression vectors for gene therapy.

Abstract: The needle assembly of the invention is a quad-partite system for use with an arc-center stereotactic headframe that allows a clinician to deliver recombinant expression vectors through the needle assembly to targeted sites at exact depths in the brain. Methods for use of the needle assembly with a tube insert for the needle cannula that does not bind proteins, as in protein viral capsids, permit delivery of precise volumes of pharmaceutical compositions containing viral recombinant expression vectors for gene therapy.

Abstract: The invention provides methods for treating ocular diseases using a recombinant vehicle to express a protein useful in the treatment of ocular disease, with particular preference for use of neurotrophin-4 (NT4) for targeting subpopulations of cells in the retina. A genetically engineered gene transfer vector containing sequences encoding a growth factor such as neurotrophin-4 (NT4) is used to transduce cells of the retinal ganglion cell (RGC) layer, in situ, via administration of the vector intravitreally. Accordingly, methods are disclosed for treating subjects in need thereof by therapeutic protein delivery via a recombinant expression vector, including rescue of photoreceptors by targeting the RGC layer subpopulation of retinal cells.