gene therapy

?
  • Created by: emma
  • Created on: 22-04-13 14:46
cystic fibrosis is caused by a mutant recessive allele. it is an example of a ........ mutation.
deletion
1 of 13
what does cystic fibrosis cause?
There is a mutation in the CFTR gene. this causes a protein to be coded for which is unable to transport chloride ions across the epithelial membrane.
2 of 13
what is CFTR?
a chloride ion channel protein that transports chloride ions out of the epithelial cells. water follows by osmosis. cells are kept ,moist.
3 of 13
in custic fibrosis, the protein either isn't made or isn't functional resulting in...
dry epithelial membranes whose mucus is viscous and sticky
4 of 13
symptoms of cystic fibrosis are:
1) mucus congestion in the lungs 2)breathing difficulties and less efficient gas exchange 3) accumulation of mucus in pancreatic ducts, preventing enzymes from reaching duodenum --> fiborous cysts 4) mucus in sperm ducts --> infertility
5 of 13
what is gene replacement?
a type of gene therapy where the faulty gene is replaced by a healthy one
6 of 13
what is gene supplementation?
a type of gene therapy where one or more copies of the healthy gene are added alongside the defective one. this masks the effects.
7 of 13
what is germ line therapy?
the replacement/supplementation of the defective gene in the fertilised egg. all cells of the organism will develop normally and so will all the cells of their offspring.
8 of 13
what is somatic-cell gene therapy?
supplementation/replacement in just the affected tissues. additional gene not present in sperm/egg so not passed on to future generations. cells die and need replacing.. treaatment needs repeating periodicaly.
9 of 13
how can viruses be used to deliver cloned CFTR genes? (somatic cell therapy)
1) made harmless by interfering with replication gene 2)grown in ep. cells in lab along with plasmids with CFTR gene inserted3) CFTR becomes incorporated into virus DNA 4) viruses isolated/purified 5)introduced into nostrils5)inject DNA into ep cells
10 of 13
how can the CFTR gene be delivered to lung cells by wrapping it in lipid?
1) CFTR isolated and put into plasmid vectors 2) put back into bacterial hosts...gene markers show which have taken up gene 3) bacteria cloned--> multiple copies of CFTR plasmid4)plasmid extracted + wrapped in lipid 5) put in aerosol + cross CMS
11 of 13
why ar the forms of delivering gene therapy not always effective?
1)adenovirus MAY cause infection 2) patients may develop immunity to virus 3aerosol may not be fine enough to pass through bronchioles 4) even when delivered, few CFTR genes are expressed
12 of 13
how can gene therapy be used to treat SCID?
1)normal ADA gene isolatedusing RE 2)inserted into reterovirs 3) grown in lab to increase numbers 4)mixed with patients t cells 5)inject ADA gene into t cells 6) t cells reintroduced to blood to provide genetic code needed to make ADA
13 of 13

Other cards in this set

Card 2

Front

what does cystic fibrosis cause?

Back

There is a mutation in the CFTR gene. this causes a protein to be coded for which is unable to transport chloride ions across the epithelial membrane.

Card 3

Front

what is CFTR?

Back

Preview of the front of card 3

Card 4

Front

in custic fibrosis, the protein either isn't made or isn't functional resulting in...

Back

Preview of the front of card 4

Card 5

Front

symptoms of cystic fibrosis are:

Back

Preview of the front of card 5
View more cards

Comments

No comments have yet been made

Similar Biology resources:

DNA
See all Biology resources »See all Practical applications of biology resources »