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Ex vivo gene therapy directed to liver in a patient with familial hypercholesterolemia

✍ Scribed by M. Grossman; P.-J. Lupien; S.E. Raper; J.M. Wilson

Book ID: 118325105
Publisher: Elsevier Science
Year: 1994
Tongue: English
Weight: 310 KB
Volume: 109
Category: Article
ISSN: 0021-9150
DOI: 10.1016/0021-9150(94)93434-7

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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