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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
Q

What are pharmacological modulators?

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26
Q

What are the 2 possible targets of pharmacological modulators?

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27
Q

What are Bcl-abl kinase inhibitors?

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28
Q

Which drug can treat CF and how?

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29
Q

What is combination therapy?

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30
Q

How does CF combination therapy work?

A
31
Q

What is stop codon read through?

A
32
Q

How can drugs be used to treat diseases caused by non-sense mutations?

A
33
Q

Which drug can be used for DMD patients and how?

A
34
Q

What is an example of a pharmacological chaperone?

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35
Q

What is an example of a pharmacological modulator?

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36
Q

What is an example of a pharmacological combination therapy?

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37
Q

What is an example of a stop codon read through therapy?

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38
Q

What are the 3 approaches to treating genetic disorders?

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39
Q

What is the main concept of gene therapies?

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40
Q

What are the 3 main limitations of developing gene therapies?

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41
Q

What does in vivo mean?

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42
Q

What does in vitro mean?

A
43
Q

Are gene therapies easier to do in vitro or vivo?

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44
Q

What are the 2 types of in vitro gene therapies?

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45
Q

How is mitochondrial disease therapy done?

A
46
Q

What are 3 parent babies?

A
47
Q

What does gene silencing use?

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48
Q

How do antisense oligonucleotides work?

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49
Q

What are the 2 types of in vivo gene therapies?

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50
Q

What is ASO?

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51
Q

What is an example of an ASO gene therapy?

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52
Q

How does RNAi therapy work?

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53
Q

How RNA cleavage activated?

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54
Q

What is an example of RNAi gene silencing?

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55
Q

What is viral gene therapy?

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56
Q

What are 4 examples of viruses used for viral gene therapy?

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57
Q

How can SCID be treated?

A
58
Q

What are the … streps of ADA-SCID treatment?

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59
Q

What does Libmeldy treat and how?

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60
Q

What is an example of a gene therapy for MLD?

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61
Q

What is an example of a gene therapy for SCD?

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62
Q

What is an example of a gene therapy for β-thalassemia?

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63
Q

What is supplementation gene therapy?

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64
Q

How does in vivo supplementation therapy work?

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65
Q

What is are 3 examples of in vivo supplementation gene therapies?

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66
Q

What are 2 potential future in vivo gene therapies?

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1.4 POM Genetics (9 decks)

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