Aav4 vector and uses thereof
AssigneeThe USA, as represented by the Secretary, Dept. of Health and Human Services
InventorsJohn A. Chiorini, Robert M. Kotin, Brian Safer
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Abstract
The present invention provides an adeno-associated virus 4 (AAV4) virus and vectors and particles derived therefrom. In addition, the present invention provides methods of delivering a nucleic acid to a cell using the AAV4 vectors and particles.
Claims21
1-42. (canceled)
A nucleic acid vector comprising:
- a) a pair of inverted terminal repeats, each of which is capable of forming a T-shaped hairpin structure, and wherein at least one inverted terminal repeat comprises an AAV4 Rep protein binding site and an AAV4 terminal resolution site (trs);
- and,
- b) a heterologous nucleic acid sequence between the terminal repeats.
A recombinant AAV4 particle comprising a nucleic acid vector comprising:
- a) a pair of inverted terminal repeats, each of which is capable of forming a T-shaped hairpin structure, and wherein at least one inverted terminal repeat comprises an AAV4 Rep protein binding site and an AAV4 terminal resolution site (trs);
- and,
- b) a heterologous nucleic acid sequence between the terminal repeats.
A method for treating a subject for a disease, comprising administering to a subject in need of such treatment a recombinant AAV4 particle comprising a nucleic acid vector comprising:
- a) a pair of inverted terminal repeats, each of which is capable of forming a T-shaped hairpin structure, and wherein at least one inverted terminal repeat comprises an AAV4 Rep protein binding site and an AAV4 terminal resolution site (trs);
- and,
- b) a heterologous nucleic acid sequence between the terminal repeats;
- wherein the heterologous nucleic acid sequence is functionally linked to a promoter, and wherein the heterologous nucleic acid sequence encodes a therapeutic agent for treating the disease.
Dependencies are inferred from the claim text.