Back to results
US 11,634,472 B2Granted
Treatment of monogenetic disorders
AssigneeZentrum für Forschungsförderung in der Pädiatrie GmbH
InventorsHelmut Hanenberg, Maj-Kristin Holz, Katharina Röllecke, Katharina Waack-Buchholz
Life of this patent
Estimating the term…
Abstract
The present invention relates to a nucleic acid expression cassette, in particular for the expression of a human liver-specific and/or liver-expressed protein and/or preferably physiologically active domains and/or fragments thereof in a patient suffering from a monogenetic disorder caused by a mutation in the gene coding for the liver-specific and/or liver-expressed protein.
Claims8
A non-viral vector, comprising a nucleic acid expression cassette, wherein the expression cassette comprises:
- (a) a promoter directing gene expression in human cells;
- (b) a coding sequence containing a nucleic acid sequence coding for coagulation factor FVIII;
- (c) a nucleic acid sequence derived from the scaffold/matrix attachment region of a eukaryotic gene, wherein said nucleic acid sequence has a nucleic acid sequence according to SEQ ID NO. 11 or at least 95% identity to SEQ ID NO. 11;
- and
- (d) a transcriptional termination signal.
2 more elements
Dependencies are inferred from the claim text.