Raav expression systems for genetic modification of specific capsid proteins
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsKenneth Warrington, Shaun Opie, Nicholas Muzyczka
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AssigneeUniversity of Florida Research Foundation, Inc.
InventorsKenneth Warrington, Shaun Opie, Nicholas Muzyczka
Estimating the term…
Disclosed are improved recombinant adeno-associated viral (rAAV) vectors having mutations in one or more capsid proteins. Exemplary vectors are provided that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations that comprise them useful in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
A recombinant adeno-associated viral expression system that expresses at least a first, second, and third distinct capsid proteins, comprising:
A recombinant adeno-associated viral expression system that expresses at least a first, second, and third distinct capsid proteins, comprising:
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An rAAV vector comprising a nucleic acid segment modified to express functional VP1 and VP3 capsid proteins substantially in the absence of functional VP2 protein.
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An rAAV vector comprising:
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An rAAV vector comprising a nucleic acid segment that has been modified to express functional VP2 and VP3 capsid proteins substantially in the absence of functional VP1 protein.
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A VP2-free rAAV expression system comprising:
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An rAAV vector comprising at least a first mutation in the capsid gene, wherein said mutation substantially reduces or eliminates the affinity of a viral particle comprising said vector for binding to heparin, heparan sulfate, or heparan sulfate proteoglycan.
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Dependencies are inferred from the claim text.