Non-viral vesicle vector for cardiac specific gene delivery
OwnerKenneth Chien, Masahiko Hoshijimainventors
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Abstract
The invention is a non-viral vesicle vector for the delivery of nucleic acid to various cardiac cell types. The vesicle vector contains the hepatitis B envelope protein wherein at least part of the liver targeting sequence is deleted and replaced with a specific cardiac cell targeting sequence. The targeting sequence may be derived from viruses that have the natural tropism desired (e.g. adenovirus type 5 knob protein for cardiomyocyte delivery) or mammalian sequences (e.g. endothelin-1 for vascular endothelial cell delivery). The vesicle vector contains an expression construct for the expression of therapeutic genes in cardiac tissues.
Claims20
A non-viral vector comprising:
- a vesicular membrane with hepatitis B envelope (env) protein containing a cardiac targeting sequence exposed on the surface of the vesicle and
- a nucleic acid construct comprising a nucleotide sequence for cardiovascular gene therapy and a promoter sequence functional in cardiovascular cells.
A non-viral vesicle vector comprising:
- a vesicular membrane with hepatitis B env protein exposed on the vesicle surface and
- a protein for treatment of cardiovascular disease.
A method for treatment of cardiac disease comprising:
- intravenous administration to an individual with cardiac disease a non-viral vesicle vector comprising a vesicular membrane with hepatitis B env protein with a cardiac targeting sequence exposed on the vesicle surface and
- a nucleic acid construct comprising a nucleotide sequence for cardiac gene therapy and a promoter sequence functional in cardiac cells
- monitoring the individual for amelioration of disease.
Dependencies are inferred from the claim text.