NON-VIRAL DNA VECTORS AND USES THEREOF FOR EXPRESSING FVIII THERAPEUTICS

摘要: The application describes ceDNA vectors having linear and continuous structure for delivery expression of a transgene. comprise an cassette flanked by two ITR sequences, where the encodes transgene encoding FVIII protein. Some further cis-regulatory elements, including regulatory switches. Further provided herein are methods cell lines reliable gene protein in vitro, ex vivo using vectors. Provided method compositions comprising useful cell, tissue or subject, treatment diseases with said expressing Such can be expressed treating disease, e.g., hemophilia A.