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Flashcards in Exam 3: Extra Credit Deck (15)
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1
Q

what does mRNA do?

A

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

2
Q

proteins =

A

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

3
Q

therapeutic approach to mRNAs: siRNA

A

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)

4
Q

therapeutic approach to mRNAs: gene therapy

A

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

5
Q

moderna approach to mRNA

A

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

6
Q

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

A

true

7
Q

importance of mRNA and intracellular proteins

A

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

8
Q

post-translational modification role in mRNA

A

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

9
Q

mRNA flexibility and speed aspect comes from

A

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

10
Q

want a drug to be reliable

A

single mRNA seq encodes a single protein

11
Q

dose

A

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.

12
Q

mRNA amplification

A

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

13
Q

mRNA feature of being confined to the cytoplasm

A

do not have to worry about changes to the genome

14
Q

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

A
  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
15
Q

moderna’s mRNA vaccine approach

A

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