| abstract |
The present disclosure provides for the insertion of a sequence encoding a therapeutic alpha-galactosidase A (alpha-GalA) protein, such as a nuclease, and an enzyme into a cell, thereby providing a protein or cell therapeutic for the treatment and / or prevention of Fabry disease. Methods of using these nucleases to provide are provided. Further provided is a composition for expressing at least one alpha-GalA protein. In one aspect, there is provided a method of expressing at least one α-galactosidase A (α-Gal A) protein in a cell, the method comprising at least one of the above α-Gal A proteins being expressed in the cell. administering a GLA transgene encoding an α-Gal A protein to the cells. |