The subject invention concerns materials and methods for gene therapy. One aspect of the invention pertains to vectors which can be used to effect genetic therapy in animals or humans having genetic disorders where expression of high levels of a protein of interest are required to treat or correct the disorder. The subject invention also pertains to methods for treating animals or humans in need of gene therapy to treat or correct a genetic disorder. The materials and methods of the invention can be used to provide therapeutically effective levels of a protein that is non-functional, or that is absent or deficient in the animal or human to be treated. In one embodiment, the materials and methods can be used to treat alpha-1-antitrypsin deficiency.
申请公布号
WO9955564(A8)
申请公布日期
1999.12.02
申请号
WO1999US08921
申请日期
1999.04.23
申请人
UNIVERSITY OF FLORIDA;FLOTTE, TERENCE, R.;SONG, SIHONG;BYRNES, BARRY, J.;MORGAN, MICHAEL
发明人
FLOTTE, TERENCE, R.;SONG, SIHONG;BYRNES, BARRY, J.;MORGAN, MICHAEL