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11 Adeno-associated virus vector-mediated gene therapy in a murine model of mucopolysaccharidosis type I

โœ Scribed by Lalitha Belur; Zachary deMorest; Chester B. Whitley; Walter C. Low; R. Scott McIvor


Book ID
116988068
Publisher
Elsevier Science
Year
2007
Tongue
English
Weight
58 KB
Volume
92
Category
Article
ISSN
1096-7192

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## Abstract ## Background Familial hypercholesterolemia is an inherited disease caused by mutations in the LDL receptor gene leading to severe hypercholesterolemia and atherosclerosis. The LDL receptor is predominantly expressed in the liver, making it a preferred target organ for somatic gene the