CPC C12N 15/86 (2013.01) [C07K 14/755 (2013.01); C12N 2750/14143 (2013.01)] | 13 Claims |
1. A capsid-free system capable of producing closed-end DNA (ceDNA) comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette;
wherein the first ITR and the second ITR are ITRs of an erythrovirus parvovirus B19 or a goose parvovirus (GPV), wherein the genetic cassette is positioned between the first ITR and the second ITR, and wherein the genetic cassette encodes a Factor VIII (FVIII) protein comprising an amino acid sequence at least 90% identical to the amino acid sequence of SEQ ID NO: 106 or 109.
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