09.30.1 Molecular Therapeutics

Front 1

natural product chemistry

Back 1

IDing the active ingredient in natural products and making that into medicine

Front 2

rational drug design

Back 2

IDing a target for drug therapy, then working out a drug from there. usually involves modification of an natural hormone/neurotransmitter (like making antihistamine from histamines)

Front 3

how to make recombinant proteins

Back 3

- insert desired gene into plasmid or virus vector to make recombinant DNA
- transform plasmid into bacteria/insect/yeast cell
- transfect plasmid into human cell
- transduce virus vector into human cells
- purify the protein made by bacteria/insect/yeast/human cell

Front 4

polycythemia

Back 4

- too much erythrocytes = too much red blood cells
= viscus blood = heart failure

Front 5

antibodies

Back 5

- proteins that bind specifically to other proteins or molecules
- usually used by body to tag infectious things for destruction
- can also be used to tag extra proteins in body for destruction

Front 6

chorionic gonadotrophin hormone

Back 6

- The hormone that preg. test antibodies test for to see if your pregers

Front 7

monoclonal antibodies

Back 7

- antibodies that come from single immortalized mouse cell
- quickly destroyed by our immune sys b/c they are seen as foreign bodies

Front 8

Humanized monoclonal antibodies

Back 8

- monoclonal antibodies that are still made in mice, but are altered to look human to immune sys

Front 9

Erythropoietin (EPO)

Back 9

how it works:
- low O2 in tissues tells Kidney to make EPO
- EPO is a glycoprotein hormone that tells bone marrow to make RBC
- RBC carries O2 to tissues
recombinant protein form is used to treat anemia in chemo patients

Front 10

Herceptin

Back 10

- ErbB2 binds to ErbB2 receptors and causes receptor dimerization = promotes cell division. is over expressed in some breast cancers.
- Herceptin is humanized monoclonal antibody that binds ErbB2/HER2/neu receptor and prevents ErbB2/HER2/neu from binding to receptor
- has side effects like heart attack, so shouldn't be used by people who don't have ErbB2 over expression

Front 11

Disease that can be treated with Gene Therapy since they are caused by a single gene

Back 11

- severe combined immune deficiency (SCID)
- hemophilia
- cystic fibrosis
- sickle cell
- musclar dystrophy

Front 12

severe combined immune deficiency (SCID)

Back 12

- bubble body disease
- can be treated with bone marrow transplant (but hard to find doner) or gene therapy

Front 13

viral vectors for gene therapy have:

Back 13

- packaged DNA that is long -lived and stable
- mutations added to make it not infectious
- helper viruses to help it replicate

Front 14

Lentiviruse

Back 14

- a retrovirus used for gene therapy (retro - has RNA that is reverse transcribed into DNA, and then inserted into host DNA)
- advantage: permanent treatment
- disadvantage: risk of cancer b/c that DNA could insert anywhere (to turn on oncogene or turn off tumor suppressor)

Front 15

Adenoviruses

Back 15

- DNA virus that causes colds/eye infections
- advantage: no integrated into host
- disadvantage: treatment has to be taken over and over. This leads to immune response that can kill patient

Front 16

Adeno-associated virus

Back 16

advantage:
- integrated into host genome = permanent treatment = no immune response
- integrated at specific site = less risk of cancer
- infects lots of diff cell types
Disadvantage
- can only package small amount of DNA
Example: Glybera

Front 17

siRNA

Back 17

- small sections of RNA that cuts specific sequences in mRNA
- sometimes targets wrong thing
- treats Ebola and blindness (due to age related macular degeneration)
- This is still just theoretical

Front 18

antisense RNA

Back 18

- binds to mRNA and inhibits expression
- example: Fomivirsen/Vitravene