Abstract: Targeted non-surgical administration of a nucleic acid formulation to the suprachoroidal space (SCS) of the eye of a human subject permits effective treatment of ocular disorders, including posterior ocular or choroidal maladies. In one embodiment, the method comprises inserting a hollow microneedle into the eye at an insertion site and infusing a nucleic acid formulation through the inserted microneedle and into the suprachoroidal space of the eye. The infused nucleic acid formulation flows within the suprachoroidal space away from the insertion site. In one embodiment, the fluid nucleic acid formulation comprises nucleic acid nanoparticles consisting of one molecule of nucleic acid.

Abstract: A method of providing a therapeutic effect in a human patient which comprises administering to the patient CD34+ cells obtained from cord blood. The CD34+ cells have been engineered with at least one nucleic acid sequence encoding a therapeutic agent. Such CD34+ cells may be engineered by transducing the cells with a retroviral vector including the nucleic acid sequence encoding the therapeutic agent. This method has been applied in treating newborn infants suffering from ADA deficiency.