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5. Adeno-associated virus mediated gene therapy in a murine model of Morquio syndrome type A

โœ Scribed by Carlos Almeciga; Adriana Montaqo; Luis Barera; Shunji Tomatsu


Book ID
116988440
Publisher
Elsevier Science
Year
2009
Tongue
English
Weight
44 KB
Volume
96
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