The mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders in which the storage material is glycosaminoglycan. Each MPS is caused by the genetic deficiency of a single lysosomal enzyme. Due to the nature of these diseases and the characteristics of the enzymes that are deficient most of the MPS are goodcandidates for gene therapy. Studies in animal models have supported this contention and have shown that several different approaches to gene therapy for the MPS are possible. However, it is also clear that each of these approaches is limited by the currently available technology and that the development of new gene delivery technology is a priority.
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