Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 7, Issue 9, Pages 1333-1345Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1517/14712598.7.9.1333
Keywords
gene therapy; glycosaminoglycan; lysosomal storage disease; mucopolysaccharidosis
Funding
- NATIONAL CENTER FOR RESEARCH RESOURCES [P40RR002512] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES [R01DK066448, R01DK025759, R01DK054481, R56DK066448] Funding Source: NIH RePORTER
- OFFICE OF THE DIRECTOR, NATIONAL INSTITUTES OF HEALTH [P40OD010939] Funding Source: NIH RePORTER
- NCRR NIH HHS [RR02512, P40 RR002512] Funding Source: Medline
- NIDDK NIH HHS [DK25759, R56 DK066448, R01 DK054481, R01 DK066448, DK066448, DK54481, R01 DK025759, R01 DK054481-16] Funding Source: Medline
- NIH HHS [P40 OD010939] Funding Source: Medline
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Mucopolysaccharidoses (MPS) are due to deficiencies in activities of lysosomal enzymes that degrade glycosaminoglycans. Some attempts at gene therapy for MPS in animal models have involved intravenous injection of vectors derived from an adeno-associated virus (AAV), adenovirus, retrovirus or a plasmid, which primarily results in expression in liver and secretion of the relevant enzyme into blood. Most vectors can correct disease in liver and spleen, although correction in other organs including the brain requires high enzyme activity in the blood. Alternative approaches are to transduce hematopoietic stem cells, or to inject a vector locally into difficult-to-reach sites such as the brain. Gene therapy holds great promise for providing a long-lasting therapeutic effect for MPS if safety issues can be resolved.
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