Recombinant adeno-associated virus gene therapy vectors with reduced liver tropism and enhanced transduction of cardiac cells for the therapy of heart diseases and diseases associated with heart dysfunction
AssigneeAavigen GmbH
InventorsMartin BUSCH, Patrick MOST, Gregor HABECK, Jörg SCHWEIGGERT
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Abstract
The present invention provides recombinant adeno-associated virus vectors which show cardiac enrichment but diminished enrichment in other organs, in particular liver and CNS cells, for the treatment of acquired and hereditary left- and right-sided heart diseases as well as other diseases directly or indirectly associated with heart dysfunction.
Claims26
A recombinant adeno-associated virus (rAAV) vector, comprising a nucleic acid molecule encoding a capsid protein and a heterologous nucleic acid sequence encoding a gene product of interest, wherein the nucleic acid molecule encoding the capsid protein comprises a sequence at least 90% identical to SEQ ID NO:
- 22, 28, 195, 197 or 199, and wherein the rAAV vector has tropism for human cardiomyocytes.
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A rAAV capsid protein comprising a sequence selected from the group consisting of SEQ ID NOs:
- 99, 105, 196, 198 and 200.
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Dependencies are inferred from the claim text.