gene therapy

Question 1

define gene therapy

Answer 1

changing faulty alleles that cause genetic disease

Question 2

for dominant alleles:
what is sufferer?
need to…

Answer 2

• sufferer heterozygous
• need to silence dominant allele
• use vector to add DNA fragment into dominant allele to prevent it from being transcribed

Question 3

for recessive alleles:
what is sufferer?
need to…

Answer 3

• sufferer homozygous

- use vector to add functional (dominant) allele to DNA

Question 4

germ line gene therapy vs somatic gene therapy

Answer 4

germ line changes alleles of gametes so all future offspring inherit change whereas somatic therapy changes the alleles of body cells so change is not permanent

Question 5

give 4 potential issues

Answer 5

• allele inserted into wrong locus
• wrong gene silenced (i.e tumour supressor gene)
• non-medicinal uses
• gene overexpressed