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1.4 POM Genetics > Emerging Treatments > Flashcards

Emerging Treatments Flashcards

1
Q

What is the most common type of genetic disorder?

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2
Q

What are 4 examples of genetic disorders caused by errors in metabolism?

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3
Q

What is PKU?

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4
Q

What are the 4 symptoms of untreated PKU?

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5
Q

How is PKU treated and why?

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6
Q

What is haemophilia?

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7
Q

What are the 3 symptoms of haemophilia?

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8
Q

How is haemophilia treated?

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9
Q

What are 4 examples of diseases treated using replacement via diet?

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10
Q

How is growth hormone deficiency treated?

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11
Q

How is lysosomal storage disease treated?

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12
Q

How is Fabry disease treated?

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13
Q

How is Pompe disease treated?

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14
Q

What knowledge is required to design treatments via diet?

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15
Q

What are pharmacological treatments?

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16
Q

What do pharmacological treatments usually target?

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17
Q

What are the 4 stages of drug development?

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18
Q

What is the NICE?

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19
Q

Which 2 organisations approve drugs for usage in the UK?

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20
Q

Do pharmacological therapies targeting proteins cure the disease?

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21
Q

What are the 4 types of pharmacological therapies?

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22
Q

What are pharmacological chaperones?

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23
Q

How do pharmacological chaperones work?

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24
Q

Which drug can be used to treat Fabry disease and why?

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25
What are pharmacological modulators?
26
What are the 2 possible targets of pharmacological modulators?
27
What are Bcl-abl kinase inhibitors?
28
Which drug can treat CF and how?
29
What is combination therapy?
30
How does CF combination therapy work?
31
What is stop codon read through?
32
How can drugs be used to treat diseases caused by non-sense mutations?
33
Which drug can be used for DMD patients and how?
34
What is an example of a pharmacological chaperone?
35
What is an example of a pharmacological modulator?
36
What is an example of a pharmacological combination therapy?
37
What is an example of a stop codon read through therapy?
38
What are the 3 approaches to treating genetic disorders?
39
What is the main concept of gene therapies?
40
What are the 3 main limitations of developing gene therapies?
41
What does in vivo mean?
42
What does in vitro mean?
43
Are gene therapies easier to do in vitro or vivo?
44
What are the 2 types of in vitro gene therapies?
45
How is mitochondrial disease therapy done?
46
What are 3 parent babies?
47
What does gene silencing use?
48
How do antisense oligonucleotides work?
49
What are the 2 types of in vivo gene therapies?
50
What is ASO?
51
What is an example of an ASO gene therapy?
52
How does RNAi therapy work?
53
How RNA cleavage activated?
54
What is an example of RNAi gene silencing?
55
What is viral gene therapy?
56
What are 4 examples of viruses used for viral gene therapy?
57
How can SCID be treated?
58
What are the ... streps of ADA-SCID treatment?
59
What does Libmeldy treat and how?
60
What is an example of a gene therapy for MLD?
61
What is an example of a gene therapy for SCD?
62
What is an example of a gene therapy for β-thalassemia?
63
What is supplementation gene therapy?
64
How does in vivo supplementation therapy work?
65
What is are 3 examples of in vivo supplementation gene therapies?
66
What are 2 potential future in vivo gene therapies?

1.4 POM Genetics (9 decks)

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