gene therapy tissue engineering

Question 1

gene therapy

Answer 1

taking a copy of healthy gene and finding an effective way of getting it into the cells that need it, so they can produce the correct protein

Question 2

how does cystic fibrosis affect respiratory system and symptoms

Answer 2

thick mucus builds up reduces flow to alveoli
coughing fits
breathless

Question 3

how is dmd caused

Answer 3

one or more deletions in the dystrophin gehe

Question 4

gene silencing/exon skipping

Answer 4

• drug that is complimentary to mutated sequence (drisapersen)
• binds to mrna over the deletion so becomes double stranded
  -so that bit is not translated
• restores reading frame so shorter dystophin produced

Question 5

somatic cell therapy

Answer 5

• therapeutic genes transferred into body cells of patient
• genetic changes are are not inherited by gamete’s
• dna introduced by vector

Question 6

germ line therapy

Answer 6

• sperm or eggs are modified by the introduction of functional genes
• heritable
  but rare due to ethical reasons

Question 7

effectiveness of gene therapy

Answer 7

• plasmid not always taken up
• if it is taken up sometimes gene isn’t always expressed

Question 8

gene therapy for CF

Answer 8

• healthy CFTR gene into liposome
• inhaled by lungs
• fuse with cm and pass dna into cytoplasm
• only treatment has to be repeated

Question 9

liposome

Answer 9

a hollow phospholipid sphere used as a vehicle to carry molecules into a cell

Question 10

cells are seeded onto a scaffold- an artificial structure that can support a 3d tissue
they must allow

Answer 10

-diffusion of nutrients and waste products
-allow cells to attach and move
-be biodegradable at the same rate of surrounding tissue