Genetic Modifications Flashcards

1
Q

Describe the exceptional case with immunity from HIV

A

In 2006, there was the first patient with leukemia who is HIV+ who got bone marrow transplant and recovered. The bone marrow donor has a mutation which means that they do not possess a receptor that HIV attacks

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2
Q

What happens if you perform gene editing on a 8-cells embryo?

A

The mutation is inherited by any forthcoming cell, tissue, organ, system

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3
Q

What did geneticist He Jiankui do?

A

He performed gene editing on 2 embryos from an HIV positive parents to prevent the babies from transmission. He did this by removing the receptors which HIV uses to infect a cell.

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4
Q

When did geneticist He Jiankui conduct his experiment?

A

November 2018, Shengzou university

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5
Q

What consequences did geneticist He Jiankui face?

A

He was fired from the univeristy and condemned to 3 years of jail, since gene editing on regular embryos is not allowed.

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6
Q

What does prevention include?

A

Includes reproductive programs, preimplantation genetic diagnosis and gene testing

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7
Q

What does tertiary prevention include?

A

Includes preventing/ delaying complications of the disease

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8
Q

What are functional placements?

A

When technology is able to assist with underdevelopment due to a gene mutation

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9
Q

Define classical gene therapy

A

Delivering an exogenous copy of cDNA to replace the lost endogenous gene expression by the use of adeno-associated virus (AAV)

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10
Q

When was the first classical gene therapy done?

A

In the 1970s to produce insulin in bacteria

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11
Q

Pros of classical gene therapy

A

No risks associated with cutting the genome

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12
Q

Cons of classical gene therapy

A

Insertional mutagenesis leading to unexpected DNA mutations
Non-physiological expression of the therapeutic gene/ vector immunogenicity

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13
Q

What does CRISPR stand for?

A

Clustered Regularly Interspaced Short Palindromic Repeats

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14
Q

Where was CRISPR Cas9 evolved?

A

Some bacteria have evolved their immune system to identify these viral genomes and prevent replication

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15
Q

What is needed for CRISPR-Cas9?

A

A detector: CRISPR guide RNA
a scissor Cas-9
a therapeutic gene
vector

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16
Q

How can CRISPR_Cas9 be targeted?

A

Injected from a cell from your body modified and then injected back into the body [ex-vivo]
injected directly into the target cell [in vivo]

17
Q

Pros of Germline gene therapy

A

Reducing transmission to next generations and curing diseases prior to birth so it reduces the morbidity rate which have high costs for the healthcare system

18
Q

What are risks of germline gene therapy?

A

artificially induced mutations
few clinical trials
damaging other genes
off target cuts

19
Q

Compassionate care

A

Using sick volunteers with no alternatives

20
Q

What was He-Jiankui’s fault?

A

He should have started on a disease with no alternatives; in HIV there is an alternative such as anti-retroviral therapy

21
Q

Cons of germline editing

A
  1. Intervention costs a lot: takes away from other parts of health care
  2. Create genetic abnormalities
  3. Enhance racism