Journal
MOLECULAR THERAPY
Volume 13, Issue 5, Pages 839-849Publisher
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymthe.2006.01.006
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Funding
- NICHD NIH HHS [HD33531] Funding Source: Medline
- NIDDK NIH HHS [DK05786] Funding Source: Medline
- NINDS NIH HHS [NS34568, NS043205] Funding Source: Medline
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Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.
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