Molecular Medicine II Flashcards Preview

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Flashcards in Molecular Medicine II Deck (19):
1

what prerequirstes need to be given for gene therapy to work

-mutatio nhas to be recessible
-cells in question must be accessible to manipulation

2

what is the biggest obstable to successful gene therapy

gene delivery, you have to figure out how to insert a fragment of DNA into the chromosomal DNA of billions of cells

3

how do you do gene dlivery

contruct viral or non-viral vectors

4

what are properties that a vector needs to ahve

aggressive spread
effective integration of viral NA into host genome

5

how do you do gene delivery if you aren't using a virus

liposomes
naked DNA
complexed DNA
artifical chromsomes

6

advantages of using liposomes

easy to use, easy to find, can accommodate large DNA molecules

7

disadvantage of using liposomes

low integration rate of DNA Into genome

8

advantage of using naked DNA

easy to prepare in large amounts, no size limitation

9

disadvantages of using naked DNA

quickly degraded, entry itno cells in inefficent

10

advatanges of complexed DNA

more stable than naked DNA

11

disadvantages of complexed DNA

entry of DNA Into cells in infeffient

12

advantages of artifical chromsomes

no insertion into genome required for stable expression, no size limit

13

disadvantgae of artifical chromeomes

unpredictable chromosomal events happen durin mitosis

14

ex vivo gene therapy

-remove cells from pt
=manipulate the cells in a lab
-reimplant the modified cells

15

what is safer, in vivo or ex vivo

ex vivo

16

in vivo therapy works best on what kind of cells

epithelial

17

in vivo therapy is currently being developed as a treatment for waht disease

cystic fbirosis

18

what does herceptin treat

ErbB2 overexpresssing breast cancer

19

what does ErBb2 encorde

epidermal growth factor receptor