Journal
JOURNAL OF GENE MEDICINE
Volume 6, Issue 5, Pages 481-506Publisher
WILEY
DOI: 10.1002/jgm.581
Keywords
lysosomal storage disease; animal models; gene therapy
Categories
Funding
- NCRR NIH HHS [5T32RR007063, 5P40RR002512] Funding Source: Medline
- NIDDK NIH HHS [DK066448, DK54481, DK49652, DK25759, DK38795] Funding Source: Medline
- NIH HHS [P40 OD010939] Funding Source: Medline
- NINDS NIH HHS [K08NS-02032] Funding Source: Medline
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There are more than 40 different forms of inherited lysosomal storage diseases (LSDs) known to occur in humans and the aggregate incidence has been estimated to approach I in 7000 live births. Most LSDs are associated with high morbidity and mortality and represent a significant burden on patients, their families, and health care providers. Except for symptomatic therapies, many LSDs remain untreatable, and gene therapy is among the only viable treatment options potentially available. Therapies for some LSDs do exist, or are under evaluation, including heterologous bone marrow transplantation (BMT), enzyme replacement therapy (ERT), and substrate reduction therapy (SRT), but these treatment options are associated with significant concerns, including high morbidity and mortality (BMT), limited positive outcomes (BMT), incomplete response to therapy (BMT, ERT, and SRT), life-long therapy (ERT, SRT), and cost (BMT, ERT, SRT). Gene therapy represents a potential alternative therapy, albeit a therapy with its own attendant concerns. Animal models of LSDs play a critical role in evaluating the efficacy and safety of therapy for many of these conditions. Naturally occurring animal homologs of LSDs have been described in the mouse, rat, dog, cat, guinea pig, emu, quail, goat, cattle, sheep, and pig. In this review we discuss those animal models that have been used in gene therapy experiments and those with promise for future evaluations. Copyright (C) 2004 John Wiley Sons, Ltd.
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