Methods for the production of chimeric adeno-associated virus (AAV) vectors, compositions of chimeric AAV vectors, and methods of use thereof

摘要

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.