Gene therapy and Macromolecule tissue engineering

Question 1

What is Tissue Engineering?

Answer 1

The understanding of mammalian tissues and how to restore, maintain or improve tissue function using biological substituents

Question 2

What are the different gene therapy vectors?

Answer 2

Viral
Synthetic
plasmid
naked DNA

Question 3

What are some diseases that are targets for gene therapy?

Answer 3

Cystic fibrosis - Caused by the CTFR gene missing in the lung, gut and biliary tree (liver, gallbladder and bile ducts). Generates thick mucus

Sickle cell anaemia

Haemophilia - Clotting does not happen properly. If they get cut, they will bleed continuously, as different factors such as factor 8 is
missing. treatment is to introduce the factor into the body.

OTCD - Prevents metabolism of ammonia. Ammonia is naturally generated in the
body.