- Madrid, ES Jose Carlos Segovia Sanz - New York NY, US Africa Gonzalez Murillo - New York NY, US Jose Antonio Casado Olea - New York NY, US Guillermo Güenechea Amurrio - New York NY, US
The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.