aav9-mediated gene therapy for the treatment of type i mucopolysaccharidosis

摘要

a co-therapeutic regimen comprising intravascular / intracisternal and / or systemic aav9 administration of an expression cassette containing a hydua gene and two or more immunosuppressants is provided. useful methods are also provided for the treatment of water deficiency (mpsi) and symptoms associated with hurler, hurler-scheie and scheie syndromes.