Abstract: Provided are adeno-associated virus (AAV) compositions that can restore IDS gene function in cells, and methods for using the these AAV compositions to treat disorders associated with reduction of IDS gene function (e.g., Hunter syndrome). Also provided are compositions, systems and methods for making the AAV compositions.

Abstract: A viable global NaV1.7?/? knockout mouse is disclosed, and a breeding colony of global NaV1.7?/? knockout mice. Also disclosed are an isolated mouse gamete that does not encode a functional NaV1.7?/?, produced by the NaV1.7?/? knockout mouse; an isolated NaV1.7?/? mouse cell, or a progeny cell thereof, isolated from the NaV1.7?/? knockout mouse; and a primary cell culture or a secondary cell line and a tissue or organ explant or culture thereof derived from the NaV1.7?/? knockout mouse. Disclosed also are a hybridoma, wherein the hybridoma was originally formed from the fusion of the isolated NaV1.7?/? mouse cell mouse cell and a myeloma cell, and a method of making an antibody. Also disclosed are assays useful for screening prospective NaV1.7 inhibitors and dose ranging a test NaV17 inhibitor compound, which were validated using the NaV1.7?/? knockout mouse.