Lecture 5

Question 1

What is gene therapy? Define?

Answer 1

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Question 2

What are the types of gene therapy? What are the properties of each?

Answer 2

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Question 3

What is nature’s version of gene therapy?

Answer 3

saasddas

Question 4

What needs to be considered to make gene therapy successful?

Answer 4

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Question 5

How to get DNA into cells? What methods were tried? What are the advantages and disadvantages?

Answer 5

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Question 6

What is naked DNA- to get DNA into cells? Properties? Success?

Answer 6

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Question 7

How were liposomes used to get DNA into cells? Properties? Success?

Answer 7

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Question 8

What are viral vectors? How do they help get DNA into cells?

Answer 8

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Question 9

What are the subdivisions? Which viral vectors go into these divisions? Properties?

Answer 9

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Question 10

What is a viral titre? How is it made? What is the end product?

Answer 10

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Question 11

What are integrating viral vectors? RNA/DNA? What are the types?

Answer 11

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Question 12

What is the process of integrating viral vectors?

Answer 12

dasadasda

Question 13

What is a negative aspect of the integrating viral vectors?

Answer 13

edasdada

Question 14

What are the non-integrating viral vectors? RNA /DNA? What are the types?

Answer 14

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Question 15

What are the benefits? What are the disadvantages of the different types?

Answer 15

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Question 16

What is the process of gene transfer?

Answer 16

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Question 17

What is conditioning?

Answer 17

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Question 18

What is a good place for in vivo gene transfer? What are the properties that make it a good place?

Answer 18

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Question 19

What is immune privileged organ?

Answer 19

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Question 20

What is an example of in vivo gene transfer?

Answer 20

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Question 21

What viruses are best for retinal diseases? Why?

Answer 21

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Question 22

What is ex-vivo gene transfer? Example?

Answer 22

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Question 23

What is Strimvelis? What is new about it? How does it work?

Answer 23

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Question 24

What is the whole process of ex-vivo gene transfer?

Answer 24

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Question 25

What is in Vivo-siRNA transfer? What is the process? What disease can it be used for?

Answer 25

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Question 26

What is a dominant negative mutation?

Answer 26

wdasadasd

Question 27

When is the phenotype of PPK seen? What stage in life? Why?

Answer 27

dasdadsa

Question 28

Summary of gene transfer? What is it used for? What are the advantageous? What are the disadvantages?

Answer 28

edasdadas

Question 29

What is gene correction?

Answer 29

edasdads

Question 30

What is the idea behind gene correction?

Answer 30

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Question 31

What is the whole process of gene correction? What factors need to be considered?

Answer 31

dasdadsas

Question 32

What revertant mosaicism? What is the process using an example?

Answer 32

edadadasd

Question 33

Where specifically does revertant mosaicism occur?

Answer 33

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Question 34

How does revertant mosaicism occur in EB?

Answer 34

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Question 35

How can we use revertant mosaicism for cell therapy in EB?

Answer 35

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