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Phar 210 > Gene Therapy > Flashcards

Gene Therapy Flashcards

1
Q

How many chromosomes do humans have?

A

22 + xy or xx

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2
Q

What is germ-line gene therapy?

A
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3
Q

What is somatic gene therapy?

A
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4
Q

What is exvivo gene therapy/

A
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5
Q

What is stable gene trasnfer?

A

Is obtained when the therapeutic gene integrates into host cell chromosomal DNA

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6
Q

When episomal vectors are used for gene transmittance what is required?

A

Original of replication

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7
Q

What is ADA?

A

Adenosine deaminase deficiency is where individuals are severely immunodeficienttherefore require bone marrow transplant or administration of the ADA protein

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8
Q

How often do individauls require ADA protein?

A

1 to 2 month intervals

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9
Q

What is familial hypercholesterolemia?

A
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10
Q

The most commonly employed approaches for cancer gene therapy influcde?

A
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11
Q

What is the bystander effect?

A
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12
Q

Cerepro what is it?

A

brain tumor treatment

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13
Q

What is the MDR-1 gnee/

A
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14
Q

What is luxturna?

A

Treatment for inherited genetic mutation that is associated with retinal dystrophy (1 time treatment), Progressive loss of peripheral and central vision

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15
Q

What gene does luxturna target?

A

RPE65 gene (Eye Target)

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16
Q

What is lymfactin?

A

Gene transfer vector carrying the human VEGF-C gene. Where it promotes the body to produce VEGFC to fix damage to area

17
Q

What are the limitations of gene therapy?

A

Short lives
Immune repsonse
Viral Vectors
Multigene disorders
May induce tumors

18
Q

What are the deliveries of non-viral tene transfer

A

Injection of naked DNA
particle bombardment
Entrapping DNA into liposomes

19
Q

What viruses can be used for gene transfer?

A
20
Q

What are the ideal vector for gene transfer?

A
21
Q

What is Lyfengia?

A

Cell based gene therapy that uses a lentiviral vector

22
Q

WHat is Vyjuvek?

A

Used to treat EB (Butterfly skin)

23
Q
A

Phar 210 (10 decks)

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