Exam 3: Extra Credit

Question 1

what does mRNA do?

Answer 1

produces instructions to make proteins that may treat or prevent disease (set of instructions that are not small molecules or biologics); single stranded and carries out protein synthesis

Question 2

proteins =

Answer 2

workhorse; nearly every function in the human body (normal and disease related) is carried out by protein(s)

Question 3

therapeutic approach to mRNAs: siRNA

Answer 3

siRNAs bind to and degrade specific mRNAs inside of cells ie changes the flow of information (life is the flow of information and mRNA is the working copy)

Question 4

therapeutic approach to mRNAs: gene therapy

Answer 4

DNA is introduced from the outside and translocates to into the nucleus of cells where episomal or DNA inserted into the genome of the host gets made into mRNA indirect way to increase or add a specific type of mRNA from the outside

Question 5

moderna approach to mRNA

Answer 5

modify types or amounts of mRNA inside of cells so can have a phenotypic effect in a disease. can put mRNA directly into a cell to create proteins (endogenous or foreign) as mRNA can create a full set of proteins that could treat a broad range of human diseases

Question 6

t/f: moderna is focusing on enabling targeted cells to turn on proteins that will enable the body to fight or prevent a given disease

Answer 6

true

Question 7

importance of mRNA and intracellular proteins

Answer 7

mRNA encodes every protein in the proteome ie can make any type of protein esp intracellular proteins that are not normally accessible with traditional recombinant technologies

Question 8

post-translational modification role in mRNA

Answer 8

lot of functional proteins in one’s cells act as multi-protein complexes to accomplish a job by working together. when we want to make a vaccine, the thing the immune sys needs to recognize is a multi-protein complex so we can supply all the mRNAs needed that are in that complex and the cells will construct and present it

Question 9

mRNA flexibility and speed aspect comes from

Answer 9

the 4 nucleotide bases; combination of these 4 bases create a diverse number of molecules following a standard approach that is that they always use those 4 nucleotide bases

Question 10

want a drug to be reliable

Answer 10

single mRNA seq encodes a single protein

Question 11

dose

Answer 11

ie dose dependability, want a proportional response (response goes up to incr dose in a predictable manner). see that with mRNA, the protein output is proportional to the amount of mRNA given.

Question 12

mRNA amplification

Answer 12

a feature of mRNA is that it gets amplified; one mRNA gets translated thousands of times so one mRNA can result in hundreds to thousands of proteins

Question 13

mRNA feature of being confined to the cytoplasm

Answer 13

do not have to worry about changes to the genome

Question 14

moderna’s beliefs about mRNA as a new category of medicines (4)

Answer 14

1. large product opportunity
2. higher probability of technical success
3. accelerated research and development timelines
4. greater capital efficiency over time vs recombinant technology

Question 15

moderna’s mRNA vaccine approach

Answer 15

injecting mRNA into the muscle, instead of protein, to make an (antigenic protein) and elicit an immune response (antibody and T cell responses) key is stabilizing mRNA and getting cell to take it up to make the protein