Molecular Medicine II Flashcards
what prerequirstes need to be given for gene therapy to work
- mutatio nhas to be recessible
- cells in question must be accessible to manipulation
what is the biggest obstable to successful gene therapy
gene delivery, you have to figure out how to insert a fragment of DNA into the chromosomal DNA of billions of cells
how do you do gene dlivery
contruct viral or non-viral vectors
what are properties that a vector needs to ahve
aggressive spread
effective integration of viral NA into host genome
how do you do gene delivery if you aren’t using a virus
liposomes
naked DNA
complexed DNA
artifical chromsomes
advantages of using liposomes
easy to use, easy to find, can accommodate large DNA molecules
disadvantage of using liposomes
low integration rate of DNA Into genome
advantage of using naked DNA
easy to prepare in large amounts, no size limitation
disadvantages of using naked DNA
quickly degraded, entry itno cells in inefficent
advatanges of complexed DNA
more stable than naked DNA
disadvantages of complexed DNA
entry of DNA Into cells in infeffient
advantages of artifical chromsomes
no insertion into genome required for stable expression, no size limit
disadvantgae of artifical chromeomes
unpredictable chromosomal events happen durin mitosis
ex vivo gene therapy
-remove cells from pt
=manipulate the cells in a lab
-reimplant the modified cells
what is safer, in vivo or ex vivo
ex vivo
