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AssigneeUniversity of Florida Research Foundation, Inc.
InventorsOleksandr Kondratov, Anatolii Mamchur, Maria Ukhanova, Sergei Zolotukhin
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Disclosed herein are recombinant AAV variant (e.g., variant serotype 5 (AAV5)) capsid proteins and variant capsid protein-containing viral particles with enhanced ability to transduce adult stem cells and neurons. Viral particles containing these capsid variants are capable of enhanced transduction of mammalian mesenchymal stem cells and neurons. The recombinant AAV5 variant proteins and viral particles disclosed herein were identified from a variant AAV5 capsid library that was engineered by making substitutions in variable regions of the capsid. Compositions of these variant AAV5 particles are provided that are useful for transducing and delivering therapeutic transgenes to cells, such as mesenchymal stem cells, and thus treat diseases and disorders pertaining to these cells.
A variant recombinant adeno-associated virus (rAAV) serotype 5 (AAV5) capsid protein comprising mutations in each of amino acid residues 377 and 378 in the wild-type AAV5 VP1 sequence of SEQ ID NO:
A variant recombinant adeno-associated virus (rAAV) serotype 5 (AAV5) capsid protein comprising either of the following sequences:
A variant recombinant adeno-associated virus (rAAV) serotype 5 (AAV5) capsid protein comprising mutations in at least two, at least three, at least four, at least five, or at least six of amino acid residues 436, 442, 443, 446, 447, and 448 in the wild-type AAV5 VP1 sequence of SEQ ID NO:
A variant recombinant adeno-associated virus (rAAV) serotype 5 (AAV5) capsid protein comprising mutations in at least two, at least three, at least four, at least five, at least six, or at least seven of amino acid residues 478, 479, 481, 484, 485, 486 and 489 in the wild-type AAV5 VP1 sequence of SEQ ID NO:
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