Methods for the production of chimeric adeno-associated virus (AAV) vectors, compositions of chimeric AAV vectors, and methods of use thereof
OwnerWeidong Xiao, Bernd Hauckinventors
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Abstract
The present invention provides methods for making and using chimeric AAV particles having broad tissue tropism. The AAV particles may be used to deliver nucleic acid sequences encoding a desired protein wherein infection by chimeric AAV particles provides means to transduce cells with the nucleic acid sequences. Such gene transduction results in production of the desired protein in a cell, and thus establishes or restores the activity of the protein in the cell. The present invention also provides pharmaceutical compositions comprising such chimeric AAV particles for use in the therapeutic treatment of patients.
Claims23
A method for making chimeric adeno-associated (AAV) particles comprising capsid proteins from different AAV serotypes, said method comprising:
- a) providing a vector construct wherein said vector construct encodes nucleic acid sequences encoding a polypeptide;
- b) providing a helper virus construct wherein said helper virus provides accessory functions for production of viral particles;
- c) combining at least two AAV helper constructs to produce an AAV helper construct mixture;
- d) introducing said vector construct, said helper virus construct, and said AAV helper construct mixture into a recipient cell to effect production of chimeric AAV particles capable of infecting diverse cell types.
A method for making chimeric adeno-associated (AAV) particles comprising capsid proteins from two different AAV serotypes, said method comprising:
- a) providing a vector construct wherein said vector construct encodes nucleic acid sequences encoding a polypeptide;
- b) providing a helper virus construct wherein said helper virus provides accessory functions for production of viral particles;
- c) selecting a first AAV helper construct comprising nucleic acids encoding a first capsid protein;
- d) selecting a second AAV helper construct comprising nucleic acids encoding a second capsid protein;
- e) combining said first and second AAV helper constructs to produce an AAV helper construct mixture;
- f) introducing said vector construct, said helper virus construct, and said AAV helper construct mixture into a recipient cell to effect production of chimeric AAV particles capable of infecting diverse cell types.
A chimeric AAV particle comprising capsid proteins from AAV1 and AAV2 having increased tissue tropism, said particle further comprising a sequence encoding a therapeutic protein of interest.
Dependencies are inferred from the claim text.