| Literature DB >> 12459725 |
Robert J Desnick1, Edward H Schuchman.
Abstract
The past decade has witnessed remarkable advances in our ability to treat inherited metabolic disorders, especially the lysosomal storage diseases, a group of more than 40 disorders, each of which is caused by the deficiency of a lysosomal enzyme or protein. During the past few years, both enzyme replacement and enhancement therapies have been developed to treat these disorders. This review discusses the successes and shortcomings of these therapeutic strategies, and the contributions that they have made to treating lysosomal storage diseases.Entities:
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Year: 2002 PMID： 12459725 DOI： 10.1038/nrg963
Source DB: PubMed Journal: Nat Rev Genet ISSN： 1471-0056 Impact factor: 53.242