Genetically Engineered Skin Cells for the Systemic In Vivo Treatment of Deficient Enzymes, Factors or Proteins

摘要

A method for the systemic delivery of an enzyme to treat lysosomal storage disease of a subject is provided by creating genetically modified skin cells via topical introduction of a genetically engineered virus which delivers a nucleic acid encoding an enzyme or factor for expression by the skin cells, wherein the expressed enzyme or factor is secreted by the skin cells and is introduced into the circulatory system of the subject.