Compounds and methods to enhance rAAV transduction

a technology of enhancing raav and compounding, applied in the introduction of vector-based foreign material, viruses, peptide/protein ingredients, etc., can solve the problems of major obstacles in the trafficking of internalized viruses to the nucleus, endosomal processing and other problems, to achieve the effect of increasing episomal stability or persistence of vectors, and increasing episomal stability
US20080213221A1Inactive Publication Date: 2008-09-04UNIV OF IOWA RES FOUND
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
UNIV OF IOWA RES FOUND
Publication Date
2008-09-04
Estimated Expiration
Not applicable · inactive patent

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Abstract

Agents and methods to alter rAAV transduction are provided.
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Description

STATEMENT OF GOVERNMENT RIGHTS

[0001] This invention was made at least in part with a grant from the Government of the United States of America (Contract No. HL51887 from the National Institutes of Health). The Government may have certain rights in the invention.BACKGROUND OF THE INVENTION

[0002] Recombinant adeno-associated virus (rAAV) has several characteristics that underscore its potential as a gene therapy vector for numerous target organs and inherited diseases. rAAV vector systems potentially offer major advantages over adenovirus and retroviruses. These include the ability of rAAV to integrate into the genome of non-dividing cells, the lack of potential immune responses since all viral genes can be deleted, and the fact that rAAV can be concentrated to high titers.

[0003] Adeno-associated virus type-2 (AAV-2) has been suggested to be a very promising vector for the gene therapy of cystic fibrosis (Conrad et. al., 1996; Flotte et al., 1993; Halbert et al., 1997). In vivo administr...

Claims

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