rAAV Expression Systems and Methods of Use

摘要: Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that fully virulent, yet lack functional VP2 protein expression. Also disclosed pharmaceutical compositions, virus particles, host cells, formulations comprise these modified vectors useful in the of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes cells tissues selected mammals, including, for example, human cells.