FT2016-002: 2016-2018

Generation of mouse lines expression the human UBE3A antisense Primary Investigator: Art Beaudet Dr. Beaudet proposed to prepare a series of humanized mouse

FT2016-001: 2016-2018

Expression of Ube3a via lentiviral vector delivery in blood cells to treat Angelman syndrome Primary Investigator: Joseph Anderson, Jill Silverman The combin

MoU-001: 2016-2018

Ube3a STUb gene therapy for the treatment of Angelman syndrome. Primary Investigator: K. Nash This project funded Agilis Biotherapeutics to work with Univers