Patent ID: 6582692
Filing Date: 2003-06-24
Classification: A61K,A61P,C12N,C12Y

Abstract:
A method of delivering a recombinant adeno-associated virus (AAV) virion to a mammalian subject to treat a lysosomal storage disease, said method comprising:(a) providing a recombinant AAV virion composition which comprises AAV virions, wherein said AAV virions comprise a nucleic acid molecule, said nucleic acid molecule comprising a gene encoding a lysosomal storage enzyme missing or defective in the subject operably linked to control elements capable of directing the in vivo transcription and translation of said gene; and (b) delivering said recombinant AAV virion directly to the bloodstream of said subject, to result in expression of said gene for at least 13 weeks at a level which reduces lysosomal storage in said mammalian subject.