final gene therapy

Question 1

gene therapy

Answer 1

therapeutic tool to treat/prevent disease

Question 2

gene replacement

Answer 2

replacing disease causing gene

Question 3

gene inhbititors

Answer 3

inactivating or knocking out disease causing gene

Question 4

gene vaccines

Answer 4

introducing a new or modified gene into the body to treat disease

Question 5

gene carriers

Answer 5

success –> depends on availability of appropriate gene vectors
viral vs nonviral

Question 6

viral

Answer 6

virus - highly evolved bio machines
virus-based vectors

Question 7

challenges in viral vectors

Answer 7

immune response: immune system to tackle wild-type infections
insertional mutagenesis: get into genome –> death

Question 8

non-viral vector

Answer 8

synthetic carrier
plasmid DNA
ex: lipids, polymers,

Question 9

cationic lipids (liposomes)

Answer 9

cationic head group
condense DNA
lipid moeity
lipofectin

Question 10

cationic polymers

Answer 10

polymers with primary, secondary, tertiary amine groups

Question 11

viral gene delivery

Answer 11

advantages: high gene delivery effieciency
disadvantage: immune recognition, genomic insertion, payload size limit, negative perceptions by regulatory bodies

Question 12

non-viral gene delivery

Answer 12

advantages: safer than viral systems, easy to modify
disadvantages: much less efficient gene delivery, either high transfection efficiency or low efficiency with safety