Gene and Protein Engineering in Clinical Medicine

Question 1

Production of Proteins for Use in Treatment of Disease The following goals must be achieved?

Answer 1

1. Isolation of the gene (cDNA)
2. Introduction of the gene into a host cell system where
   it is expressed
3. Growth of host cells on a commercial scale 4. Purification of product to meet rigorous standards
4. Extensive testing to establish clinical efficacy
5. Approval from regulatory authorities for clinical use

Question 2

Example of host cells ?

Answer 2

➢ Bacteria, yeast or

➢ Cultured insect or mammalian cells

Question 3

Choice of host cells depends on what?

And what is the difference between Mammalian ,Bacteria and yeast ?

Answer 3

Choice depends largely on properties of the
protein and particular features of each host cell system.

Bacteria and yeast are easier to grow than
mammalian cells.
• Mammalian cells handle expression of
larger secreted proteins better than bacteria
or yeast.

Question 4

What Medically Useful Proteins

?

Answer 4

Recombinant DNA and expression vectors
have been used to make medically useful proteins that would otherwise have been difficult to obtain in necessary quantities.

Question 5

What is the TPA ?

Answer 5

produced by cells lining the blood vessels (enzyme)

converts plasminogen, found
normally in the blood, into plasmin, a protein
that dissolves clots.

Question 6

What is the Human Growth Hormone ?

Answer 6

*Synthesized and secreted by the pituitary at the base
of the brain
• Regulates of growth and development

HGH is species specific; only the human
protein works.

Question 7

If there is HGH deficiency what happen ?

Answer 7

hypopituitary dwarfism Must treat early while bone

growth is possible.

Question 8

Viral Disease Therapy example ?

Answer 8

Hepatitis B

DNA Vaccines

Question 9

Synthesise of the viral protein for Hepatitis B

successful in ?.

Answer 9

Synthesise of the viral protein; failed in

bacteria, but was successful in yeast!

Question 10

Hepatitis B vaccine Tested for effectiveness in ?

Answer 10

primates and then

in human volunteers.

Question 11

Hepatitis B vaccine first use in ?

Answer 11

Australia

Question 12

Present gem of efficacy on Hepatitis B vaccine.

Answer 12

94.5% efficacy

Question 13

Hepatitis B vaccine can be effective only if ?

Answer 13

takes place before

infection

Question 14

Hepatitis B vaccine
And DNA Vaccines
Based on what ?

Answer 14

Hepatitis B vaccine
• Based on recombinant HBSAg.

DNA Vaccines
• Are based on use of plasmids containing
protein coding genes.

Question 15

How can we identify plasmids key to defending

against microbial pathogens ?

Answer 15

• Cut pathogen DNA into fragments and clone
into a plasmid to make a library.
• Divide the library into pools
. • Test pools in animals; give time to develop
response then inject pathogen.
Establishing New DNA Vaccines
▪ Assume any unaffected animal group contains
at least one “protective” plasmid from a pool. ▪ Subdivide this pool and repeat the testing cycle
until a single/few plasmids are identified that
confer protection.

Question 16

Some medically useful products of biotechnology ?

Answer 16

Table 18.1

Question 17

Plant Biotechnology Applications targeting what?

Answer 17

nutrition and the

environment.

Question 18

β-carotene is converted to what in our body ?

Answer 18

vitamin A

Question 19

Example of Adapting A Crop Plant To An Environment?

Answer 19

plants that are salt-tolerant

Question 20

They take the gene for enzymes that synthesize β-carotene

from the precursor are taken from??

Answer 20

daffodils

Question 21

They take the gene that responsible of protein that moves sodium ions into
the central vacuole were isolated from??

Answer 21

Arabidopsis

Question 22

Concerns Over Plant Biotechnology.?

Answer 22

• Genetic manipulation is an unnatural
interference in nature.
• Genetically altered foods are unsafe to eat
. • Genetically altered crop plants are dangerous
to the environment.
➢ Escape of transgenes into wild populations: ➢ if the gene for herbicide resistance made its way
into weeds
. ➢ beneficial insects killed from eating plants with B.
thuringiensis genes.

Question 23

Hepatitis B immunisation requires how many injections?

Answer 23

3 injections

over a 6 month period

Question 24

Which Vaccine did the lettuce made a and which proteins

Answer 24

Measles Vaccine

By Measles virus hemagglutinin (MV-H) protein

Question 25

Advantages of lettuce Measles Vaccine

.

Answer 25

Offers development of measles vaccine formulation that
is effective, temperature-stable, easy to administer in a
resource-poor setting and amenable to large scale
manufacture

Question 26

Example of

Answer 26

use transgenic dairy animals

that produce useful products in their milk.

Question 27

-1AT in humans inhibits what ?

Answer 27

elastase, which

hydrolyzes connective tissue.

Question 28

If there-alpha -1AT deficiency’s what will happen ?

Answer 28

Excess elastase is found on the surface of the
lungs of people with cystic fibrosis and is
partially responsible for their severe breathing
problems.

Question 29

Pompe’s disease ?. characteristic. Cause of disease .treatment?

Answer 29

diagnosed by absence of alight-glucosidase activity

Cardiac hypertrophy
Loss of muscle strength prevents infants from achieving developmental milestones such as sitting, standing, and walking

enzyme alpha glucosidase from the milk of transgenic
rabbits has been successfully used for treating infants

Question 30

Why’s to block Translation of mRNA?

Answer 30

Using Antisense RNA and siRNA