Gene Therapy Flashcards
(9 cards)
Process of gene therapy
Identify defective gene
Clone gene
Determine vector for delivery
In vivo vs ex vivo
4 challenges to gene therapy
Low expression
Reach target tissue
Regulation of gene activity
Mutagenesis (knockout)
What vector causes immune response?
Adenovirus
What vector doesn’t cause known disease / doesn’t cause immune response?
Adeno associated virus
Which vector integrates into host DNA?
Retroviruses
Does a liposome trigger an immune response?
No
What’s the disadvantage of using non integrating vectors?
Transient, so must get more doses
What is a good way to do gene therapy without eliciting an immune response (other than using a retrovirus)?
Use stem cells from person (umbilical cord, bone marrow)
Which has more long lasting effects? Adenoviruses or adeno associated viruses?
Adeno associated
