RECOMBINANT VIRAL VECTOR SYSTEM

摘要

The present invention relates to a system for replication and encapsidation of recombinant DNA fragments into virus particles comprised of adeno-associated viral (AAV) capsid proteins; said system uses a 165 basepair fragment of DNA which contains AAV inverted terminal repeat sequences and which is used to engineer expression vectors useful for gene therapy. The invention provides a means of obtaining recombinant viral stocks that may be used to treat patients suffering from genetic diseases.