Drug discovery II

Question 1

Antisense oligonucleotide

Answer 1

Blocks (m)RNA directly to reduce the protein being made from the particular mRNA

Question 2

mRNA medicine

Answer 2

1. scientists generated an mRNA sequence that codes for the virus spike protein
2. the RNA sequence which will be like a blueprint for making the spike protein is then covered with a lipid coating to help with delivery into the cell
3. Onces inside, the cells read the information in the mRNA sequence to produce millions of copies of the spike protein
4. the protein fragments spur the immune system to produce antibodies that can protect when a real virus enters the body

Question 3

Monoclonal antibodies and ADC

Answer 3

antibody is a type of protein that very specifically binds to its target and often blocks the function of its target

Question 4

Adeno-associated virus

Answer 4

Thru IV infusion, AAV vectors deliver transgene to the nucleus of liver cells to enable production of therapeutic proteins

Question 5

CRISPR/CAS9

Answer 5

Crispr enzyme contains guided molecule, healthy DNA copy, DNA cutting enzyme
a specially designed synthetic guidce molecule finds the target DNA strand
An enzyme cuts off the target DNA strand
The defective DNA strand is replaced with a healthy copy

Question 6

Stem cell therapies

Answer 6

Still in the early stages
FDA warns about stem cell therapies as some may be vulnerable to stem cell treatments that are illegal and potentially harmful