Biochem 3-15 Molecular And Cellular Therapeutics

Front 1

transgenics

Back 1

genetically modifying an organism's genome by introducing a gene from another species

Front 2

antisense drugs

Back 2

oligonucleotides w/ a sequence similar to that of the RNA you want to suppress

Front 3

siRNA (RNAi) drugs

Back 3

small inhibitory RNA molecules that prevent expression of target mRNA sequences

Front 4

in vivo

Back 4

Delivery method must be targeted to specific tissue
Methods include injection or gene gun, viral vectors
“Naked” DNA is very quickly chopped up in the body so vehicles are used (liposomes, nanoparticles, etc)

Front 5

ex vivo

Back 5

Cells are removed from a patient and the gene is introduced
Methods include liposomes, electroporation, gene guns, viral vectors, nanoparticals, etc
Cells are then transplanted back into the patient

Front 6

viral vector

Back 6

-virus that has been modified to deliver a specific gene to host cells
-Viruses inject their genetic material into the host cell as part of their life cycle
-We can manipulate viruses in the lab and introduce human genes
--The virus must be engineered to be replication defective meaning it can infect the cell but not replicate itself (produce new virions)

Front 7

stem cells

Back 7

Undifferentiated cells with the capacity for self-renewal and the ability to develop into differentiated cells

Front 8

pluripotent stem cell sources: SCNT

Back 8

-somatic nuclear transfer: cloning
-Place the nucleus from a differentiated cell into an enucleated unfertilized egg

Front 9

pluripotent stem cell sources: ANT

Back 9

-altered nuclear transfer: ANT= SCNT + manipulation to prevent development of cells into an embryo

Front 10

pluripotent stem cell sources: iPS and iPSCs

Back 10

reprogramming induced pluripotent cells

Front 11

autologous transplantation

Back 11

Patients transplanted with their own stem cells

Front 12

allogenic transplantation

Back 12

Patients transplanted with stem cells from a matched donor