Lecture 5 Flashcards
What is gene therapy? Define?
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What are the types of gene therapy? What are the properties of each?
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What is nature’s version of gene therapy?
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What needs to be considered to make gene therapy successful?
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How to get DNA into cells? What methods were tried? What are the advantages and disadvantages?
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What is naked DNA- to get DNA into cells? Properties? Success?
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How were liposomes used to get DNA into cells? Properties? Success?
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What are viral vectors? How do they help get DNA into cells?
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What are the subdivisions? Which viral vectors go into these divisions? Properties?
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What is a viral titre? How is it made? What is the end product?
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What are integrating viral vectors? RNA/DNA? What are the types?
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What is the process of integrating viral vectors?
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What is a negative aspect of the integrating viral vectors?
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What are the non-integrating viral vectors? RNA /DNA? What are the types?
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What are the benefits? What are the disadvantages of the different types?
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What is the process of gene transfer?
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What is conditioning?
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What is a good place for in vivo gene transfer? What are the properties that make it a good place?
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What is immune privileged organ?
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What is an example of in vivo gene transfer?
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What viruses are best for retinal diseases? Why?
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What is ex-vivo gene transfer? Example?
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What is Strimvelis? What is new about it? How does it work?
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What is the whole process of ex-vivo gene transfer?
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What is in Vivo-siRNA transfer? What is the process? What disease can it be used for?
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What is a dominant negative mutation?
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When is the phenotype of PPK seen? What stage in life? Why?
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Summary of gene transfer? What is it used for? What are the advantageous? What are the disadvantages?
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What is gene correction?
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What is the idea behind gene correction?
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What is the whole process of gene correction? What factors need to be considered?
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What revertant mosaicism? What is the process using an example?
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Where specifically does revertant mosaicism occur?
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How does revertant mosaicism occur in EB?
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How can we use revertant mosaicism for cell therapy in EB?
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