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Flashcards in Molecular medicine II Deck (19):
1

What are the two prerequisites for Gene therapy?

1. Mutated gene that you are replacing must be recessive
2. The cells must be accessible to manipulation

2

What is somatic gene therapy?

Repairing somatic cells of the body

3

What is germ-line gene therapy?

Repairing genes in gametes

4

How are genes delivered for gene therapy?

Viral/non-viral vector

5

What are the two main characteristics that are sought for a vector virus?

Aggressive spread (but not too much)
Effective integration into host DNA

6

What are the four, non-viral methods of gene delivery?

Liposomes
Naked DNA
Complexed DNA
Artificial chromosome

7

What are the advantages/disadvantages of using liposomes?

easy to use, commercially available
Slow, low integration

8

What are the advantages/disadvantages of using naked DNA?

easy to prep, can be large
DNA is quickly degraded

9

What are the advantages/disadvantages of using complexed DNA?

most stable than naked DNA, large
Not much more stable

10

What are the advantages/disadvantages of using artificial chromosome?

No insertion into genome required, no size limit
Unpredictable chromosomal events during meiosis

11

What are the steps of Ex vivio gene therapy?

1. Remove cells from pt
2. manipulate cells
3. reinsert back to pt

12

What is the function of adenosine deaminase? A lack of it lead to what? Why?

Break down deoxyadenosine
Immunodeficiency due to lymphocyts the toxic effects of dATP (Inhibits Ribonucleotide reductase)

13

What type of cells are best changed in in vivo gene therapy?

Epithelial cells or those readily accessible

14

What is the function of herceptin?

Antibody that binds teh HER@/Neu receptor for epidermal growth factor

15

What are the symptoms of Edward syndrome (4)?

rocker feet
Prominent occiput
Synpolydactyly
Heart defects

16

What are the symptoms of Down syndrome (3)?

Low set ears
Wide eyes
Heart defects

17

What are the symptoms of Patau sydrome(5)?

Cutia aplasia
Midline defects
Holoprosencephaly
Heart defects
club feet

18

What are the symptoms of Turner syndrome (5)?

Small stature
Skin fold
Carry angle decreased
Amenorrhea
Gonadal dysplasia

19

What are the symptoms of Klinefelter syndrome (5)?

Sparse facia hair
behavior issues
Small testes
DM
cynecomasita