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IMS > Gene Therapy > Flashcards

Gene Therapy Flashcards

1
Q

What is gene therapy?

A

Replacing bad genes with good (a genetic defect)

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2
Q

what are the 2 methods of gene therapy?

A

Somatic and germline

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3
Q

What will somatic gene therapy influence?

A

The individual

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4
Q

What will germline therapy influence?

A

Future generation illegal in most countries

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5
Q

What is a transgene?

A

A gene that has been transferred naturally by genetic engineering

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6
Q

What is the intro of transgene called?

A

Transgenesis - changes phenotype of an organism

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7
Q

What does a transgene describe?

A

a segment of DNA containing a gene sequence that has been isolated from one organism and introduced to a different one

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8
Q

What does a non-native segment of DNA retain?

A

Ability to produce RNA or protein in transgenic organism

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9
Q

What are the 2 methods of delivering transgene?

A

In vivo - in body

ex vivo- out of body

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10
Q

How is transgene delivery dictated?

A

By the nature of the disease

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11
Q

What does In vivo gene therapy try to do?

A

Directly deliver transgene to site of disease

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12
Q

What gene disorder can affect several tissue types?

A

Cystic fibrosis

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13
Q

How does gene therapy work?

A

Replaces a defective gene

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14
Q

What does current gene therapy use for treatments?

A

Gene addition

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15
Q

Transgenes dont integrate, what do they exist as?

A

Episomes

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16
Q

How are transgenes delivery into a cell?

A

Via delivery vehicle or vector

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17
Q

What are the most widely used transgene delivery vehicles?

A

Viruses

18
Q

Why are Viruses used for transgene delivery?

A

Experts at entering human cells and expressing transgenes

19
Q

What are the 3 common viruses used as delivery vehicles for transgenes?

A

Adenovirus - common cold
Retrovirus - HIV
Herpesvirus - cold sores

20
Q

What are lipid based delivery vehicles called?

A

Lipoplexes

21
Q

Out of the 3 viruses which one is the only one that integrates?

A

Retrovirus

22
Q

What is gene therapy best suited for the treatment of?

A

Single gene recessive disorders

23
Q

What is Jesse gelsingers relevance to gene therapy?

A

Lowest point - he had an x-linked genetic disease of the liver, his immune response to the adenovirus lead to multiple organ failure because of high ammonia levels of OTC

24
Q

What is X-SCID?

A

A lethal immunogenic disorder where immune response are severly compromised of IL2-RG gene

25
Q

What receptors share the IL2-RG gene?

A

IL2,4,7,9,15,21

26
Q

What does the IL2-RG mutation mean in regards to immunity?

A

Loss of interleukin functions, Loss of T cells and B cells defective reduced IgG- treatment is bone marrow transplant

27
Q

What can retrovirus gene therapy be used for?

A

Inset a functioning copy of IL2-RG gene using MLV vector into haemopoietic stem cells

28
Q

What is wrong with using IL2-RG for an MLV vector in the stem cells?

A

Retroviral integration in the LM02 promoter leads to expression of protein leukemoegenesis

29
Q

What can Adenovirus gene therapy be used for?

A

Head and neck cancer

30
Q

How does the adenovirus gain success with head and neck cancer patients?

A

Use oncolytic virus (specific target cancer) - onyx-15

31
Q

What antiviral medication can be usd to treat CMV infections? (brain tumours)

A

Ganciclovir

32
Q

What gene therapy approach can be used for melanomas?

A

Ex vivo with MART-1

33
Q

What is cystic fibrosis?

A

Faulty gene that controls movements of salt and water in and out of cells leads to increase mucus secretion

34
Q

What is the most common cystic fibrosis mutation?

A

F506del - deletes 3 nucleotides coding for phenylanine

35
Q

How can cystic fibrosis affect homozygote conditions?

A

Thicker mucous membrane
Absence of vas deference due to increase mucus thickness - infertility
Pancreatic insuffiency blockage with duct

36
Q

How can cystic fibrosis affect heterozygote conditions?

A

Increase airway reactivity and pool pulmonary function and chronic rhinosinutis

37
Q

What gene therapy helps CF?

A

Adenovirus
Liposome via nebulisation
Serpin enzyme

38
Q

What does original version of crispr do?

A

Snips dna in precise location to delete faulty genes

39
Q

What does new crispr do?

A

instead of cutting genome the cas9 eznyme latches on to whatever genes attached to

40
Q

How does CRISPR work with muscular dystrophy?

A

Team boosted second gene that produces protein utrophin than compensates in muscle growth and strength

41
Q

How does CRISPR work with acute kidney injury?

A

Targets KLOTHO (renal damage protection) and IL10 (anti inflammatory cytokine)

42
Q

What does Caveats do?

A

Target genes boosted in organs through body

IMS (53 decks)

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