7.012_Section_19-solution_key-2010 - Solution key-7.012...

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1 Solution key -7.012 Recitation 19 - 2010 Question You are investigating a method of gene therapy directed to the liver of a patient with familial hypercholesterolemia (FH). The patient, who is homozygous for the FH alleles, is a human recipient for FH gene therapy. She suffered a massive heart attack with permanent damage at the age of 16, revealing the severity of her disease. The procedure for her treatment is as follows. Fifteen percent of the patient's liver was removed. The liver tissue sample was then dissociated into individual hepatocytes (liver cells) and cultured in dishes. The cultured cells were provided with a wild-type copy of the gene that is mutated in this patient. The genetically altered hepatocytes were injected back into the patient via a vein entering the liver. After some time, the patient was given bile acid binding resins. a) Why are the patient’s liver cells specifically being targeted? 75% of the cholesterol is absorbed by the LDL-receptors located on the surface of the liver cells. Since the
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