Gene Therapy Flashcards

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1
Q

What is gene therapy?

A

The introduction of a specific gene into a patient as a therapeutic agent

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2
Q

When does using gene therapy to introduce a functional copy of a mutant gene work well?

A

For single gene disorders where a specific mutation in a specific gene is causing all the symptoms

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3
Q

Which diseases can be treated with the introduction of a functional gene copy?

A

Cystic fibrosis, metabolic disorders, immunodeficiency

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4
Q

How can the introduction of an inhibitory transgene be used as gene therapy?

A

When the overexpression of a product is what is causing disease

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5
Q

What is typically introduced as an inhibitory transgene for gene therapy?

A

A gene encoding miRNA for the faulty gene to knockdown its expression

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6
Q

What diseases are inhibitory transgenes used to treat?

A

Cancer, infections, some inherited genetic diseases

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7
Q

How do we get the gene into the cell for gene therapy?

A

Use modified viruses or viral-like vectors

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8
Q

Why do we use viruses to deliver gene therapy into human cells?

A

They have evolved to deliver genetic material into human cells, so are very good at it

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9
Q

What are 3 problems we have had with the viral vectors?

A
  1. Cause an immune response in the patients
  2. Size
  3. Genome integration
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10
Q

Why do we have problems with the size of the viral vector?

A

A large vector can carry more DNA, but is also more likely to cause an immune response

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11
Q

Why is genome integration a good thing with the viral vectors?

A

Genome integration will allow the inserted gene to be copied and passed on when the cell divides

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12
Q

Why is genome integration potentially a problem with the viral vectors?

A

The gene is integrated at random in the genome, so it could potentially end up in another gene and disrupt its function, leading to cancer

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13
Q

What are the two ways to deliver gene therapy?

A

In vivo and ex vivo

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14
Q

What does in vivo delivery of gene therapy involve?

A

The vector containing the transgene is injected directly into the patient

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15
Q

What are the challenges with in vivo gene therapy?

A

Hard to get the vector to target the correct tissue

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16
Q

Why is it easy to treat liver conditions in vivo?

A

Viral particles tend to get stuck in the liver

17
Q

What does ex vivo gene therapy involve?

A

Cells are removed from the patient and the vector containing the transgene is added to those cells in a culture. The cells are put back into the patient to repopulate the organ

18
Q

What are 2 pros of using ex vivo gene therapy over in vivo?

A
  1. We are able to monitor the progress and see if it is working and integrated into the chromosome
  2. If it starts going badly we can stop it
19
Q

What is a problem with ex vivo gene therapy?

A

There isn’t a lot of tissues that can be completely repopulated by a few cells

20
Q

What is somatic gene therapy?

A

Gene therapy that only modifies the cells of a consenting adult and is not passed on to offspring

21
Q

What is germline gene therapy?

A

Gene therapy that modifies germline cells and are passed on to offspring

22
Q

Why have we drawn the line at germline gene therapy?

A

We aren’t yet at a level where we can make super precise changes, nor do we really even understand what half of the genes in humans do. Any changes we make to germline cells will be permanently in the human gene pool