Vector for expressing α-L-iduronidase and method of...

Details Vector for expressing α-L-iduronidase and method of... Vector for expressing α-L-iduronidase and method of...

2006-08-15

2006-08-15

Woitach, Joseph (Department: 1633)

Drug, bio-affecting and body treating compositions

Whole live micro-organism, cell, or virus containing

C514S04400A, C435S320100, C536S023100, C536S023500

Reexamination Certificate

active

07090836

ABSTRACT:
A purified nucleic acid molecule capable of expressing a lysosomal enzyme, e.g., iduronidase or arylsulphatase, is provided. The molecule includes a promoter of the phosphoglycerate kinase gene to facilitate expression of the lysosomal enzyme in the brain. Expressing the lysosomal enzyme in the brain of patients with lysosomal storage diseases, such as mucopolysaccharidosis, provides treatment for the neurological aspects of these diseases. The invention also provides a method of gene therapy for lysosomal storage diseases by administering the nucleic acid molecule in the form of a vector.

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