首页>
外国专利>
METHODS TO TREAT MUCOPOLYSACCHARIDE TYPE II OR DEFICIENCY IN IDURONATE-2-SULFATASE USING A RECOMBINANT ADENO-ASSOCIATED VIRUS ENCODING IDURONATE-2-SULFATASE
METHODS TO TREAT MUCOPOLYSACCHARIDE TYPE II OR DEFICIENCY IN IDURONATE-2-SULFATASE USING A RECOMBINANT ADENO-ASSOCIATED VIRUS ENCODING IDURONATE-2-SULFATASE
A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.
展开▼