Genetics in Medicine

Medicine

Quiz 13 :

The Treatment of Genetic Disease

Quiz 13 :

The Treatment of Genetic Disease

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What is the effect of RNAi on its target?
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D

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In theory, cell transplantation using embryonic stem cells derived by nuclear transplantation will avoid what complication that is associated with transplantation?
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A

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What is the likely explanation when individuals with cystathionine-synthase deficiency do not respond to treatment by cofactor supplementation?
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C

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Why is Duchenne muscular dystrophy (DMD) a technically difficult disease to treat with gene therapy?
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The first successful cure of a genetic disease by gene therapy was achieved in a patient with which disorder?
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Treatment of familial hypercholesterolemia with cholestyramine results in which of the following?
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What modification lead to increased success of enzyme replacement therapy for Gaucher disease?
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What is the mechanism of decitabine treatment for sickle cell disease?
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How does curcumin treatment lead to a normalization of chloride transport in mice with the cystic fibrosis transmembrane conductance regulator (CFTR) DF508 mutation?
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Which of the following is a major limitation of retroviruses for use as gene therapy vectors?
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To what source of hematopoietic stem cells are recipients generally more tolerant of histoincompatibility?
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How might gentamicin be a therapy for individuals with a nonsense mutation in CFTR?
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What severe side effect was observed in one gene therapy trial for X-linked severe combined immune deficiency (SCID)?
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Current guidelines recommend which dietary guidelines for management of phenylketonuria (PKU)?
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Approximately 50% of patients with homocystinuria caused by cystathionine-synthase deficiency respond to treatment with which vitamin, the precursor to this enzyme's cofactor?
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Polyethylene glycol modification of adenosine deaminase for enzyme replacement therapy has which of the following effects?
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New approaches to therapy of sickle cell disease and β thalassemia are aimed at increasing expression of which gene?
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Why is insertional inactivation of an essential gene not likely to be an important risk in retroviral-mediated gene therapies?
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