gene therapy 2 Flashcards

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1
Q

what is transfection

A

treating a genetic disease by providing the sufferer with a copy of corrected defective gene

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2
Q

what is somatic therpay

A

when the copies of the corrected gene are inserted directly into the somatic cells of the sufferer

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3
Q

what doesn’t it prevent and why

A

it doesn’t prevent the disease being passed on to the offsprings as it doesn’t affect sperm and egg cells

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4
Q

therefore what is the negative

A

need to be repeated, only effective in the short term and not effective in the long term

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5
Q

therefore what should be targeted instead

A

stems cells and undifferentiated cells so the cells divide with corrected gene

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6
Q

what are the steps for somatic therapy

A
the cells are removed from the patient 
the required gene is isolated and inserted into plasmid whichis inserted into virus
the virus infects the person 
cells become genetically altered 
cells injected into patient
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7
Q

why are viruses good

A

as they enter the host cell and integrate their dna into the host cells dna

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8
Q

what is the strength of somatic thersapy

A

it is effective in the short term and less controversial

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9
Q

what is germ therpay

A

where the corrected gene is inserted into a fertilised egg

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10
Q

why is this permamnent

A

as all the cells in the embryo will have the corrected gene and will divide by mitosis

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11
Q

how does it occur

A

the therapeutic gene is inserted into a vector and treated in a culture and inserted in a nucleas less egg
creates genetically corrected embryo

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12
Q

what are the strengths

A

potentially more effective
all cells are accessible
not passed on

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13
Q

what are the weakness

A

safety and ethical issue like virus may cause autoimmune response

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14
Q

what is liposome therapy

A

when healthy gene is inserted into a plasmid of a bacteria and the bacterial cell is wrapped with lipid molecules formng a liposome

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15
Q

what happens to the liposomes

A

they are sprayed into the nostrils using aersoosl

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16
Q

why are they fast acting

A

as they are lipid soluble they can diffuse easily through the lipid phospholipid bilayer