Gene Therapy

Question 1

What is gene therapy?

Answer 1

Replacing bad genes with good (a genetic defect)

Question 2

what are the 2 methods of gene therapy?

Answer 2

Somatic and germline

Question 3

What will somatic gene therapy influence?

Answer 3

The individual

Question 4

What will germline therapy influence?

Answer 4

Future generation illegal in most countries

Question 5

What is a transgene?

Answer 5

A gene that has been transferred naturally by genetic engineering

Question 6

What is the intro of transgene called?

Answer 6

Transgenesis - changes phenotype of an organism

Question 7

What does a transgene describe?

Answer 7

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

Question 8

What does a non-native segment of DNA retain?

Answer 8

Ability to produce RNA or protein in transgenic organism

Question 9

What are the 2 methods of delivering transgene?

Answer 9

In vivo - in body

ex vivo- out of body

Question 10

How is transgene delivery dictated?

Answer 10

By the nature of the disease

Question 11

What does In vivo gene therapy try to do?

Answer 11

Directly deliver transgene to site of disease

Question 12

What gene disorder can affect several tissue types?

Answer 12

Cystic fibrosis

Question 13

How does gene therapy work?

Answer 13

Replaces a defective gene

Question 14

What does current gene therapy use for treatments?

Answer 14

Gene addition

Question 15

Transgenes dont integrate, what do they exist as?

Answer 15

Episomes

Question 16

How are transgenes delivery into a cell?

Answer 16

Via delivery vehicle or vector

Question 17

What are the most widely used transgene delivery vehicles?

Answer 17

Viruses

Question 18

Why are Viruses used for transgene delivery?

Answer 18

Experts at entering human cells and expressing transgenes

Question 19

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

Answer 19

Adenovirus - common cold
Retrovirus - HIV
Herpesvirus - cold sores

Question 20

What are lipid based delivery vehicles called?

Answer 20

Lipoplexes

Question 21

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

Answer 21

Retrovirus

Question 22

What is gene therapy best suited for the treatment of?

Answer 22

Single gene recessive disorders

Question 23

What is Jesse gelsingers relevance to gene therapy?

Answer 23

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

Question 24

What is X-SCID?

Answer 24

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

Question 25

What receptors share the IL2-RG gene?

Answer 25

IL2,4,7,9,15,21

Question 26

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

Answer 26

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

Question 27

What can retrovirus gene therapy be used for?

Answer 27

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

Question 28

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

Answer 28

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

Question 29

What can Adenovirus gene therapy be used for?

Answer 29

Head and neck cancer

Question 30

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

Answer 30

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

Question 31

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

Answer 31

Ganciclovir

Question 32

What gene therapy approach can be used for melanomas?

Answer 32

Ex vivo with MART-1

Question 33

What is cystic fibrosis?

Answer 33

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

Question 34

What is the most common cystic fibrosis mutation?

Answer 34

F506del - deletes 3 nucleotides coding for phenylanine

Question 35

How can cystic fibrosis affect homozygote conditions?

Answer 35

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

Question 36

How can cystic fibrosis affect heterozygote conditions?

Answer 36

Increase airway reactivity and pool pulmonary function and chronic rhinosinutis

Question 37

What gene therapy helps CF?

Answer 37

Adenovirus Liposome via nebulisation Serpin enzyme

Question 38

What does original version of crispr do?

Answer 38

Snips dna in precise location to delete faulty genes

Question 39

What does new crispr do?

Answer 39

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

Question 40

How does CRISPR work with muscular dystrophy?

Answer 40

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

Question 41

How does CRISPR work with acute kidney injury?

Answer 41

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

Question 42

What does Caveats do?

Answer 42

Target genes boosted in organs through body