Biopharm

Question 1

2 ways to alter gene expression

Answer 1

gene silencing

gene editing

Question 2

2 types of gene silencing

Answer 2

antisense oligonucleotides (ASO)
RNA interference (RNAi)

Question 3

ASO vs RNAi

Answer 3

ASO: single stranded
RNAi: double stranded

Question 4

MOA of ASO

Answer 4

neutralizes sense strand so that RNA won’t be transcribed

Question 5

MOA of RNAi

Answer 5

1. bind to dicer which cuts up the RNAi double strand (still stays double stranded, but tiny fragments)
2. dicer gives small pieces to RISC which splits it into single strands
3. new single strands find bad mRNA and cause its breadown

Question 6

gene silencing vs gene editing

Answer 6

silencing: affect translation
editing: affect transcription

Question 7

E.g. of ASO

Answer 7

Kynamro (mipomersen)
for hyperchol
Stops ApoB-100 which makes LDL

Question 8

e.g. of RNAi

Answer 8

Onpattro (patisiran)

Treats TTR amyloidosis

Question 9

2 types of gene editing drugs

Answer 9

zinc finger nucleases (ZFNs)

transcription activator-like effector nucleases (TALENs)

Question 10

ZFN vs TALEN

Answer 10

ZFN: 2 pieces of nuclease, complementary to DNA you want to change, can affect multiple regions of DNA
TALEN: specific to ONLY 1 region of DNA, more cytotoxic, bigger than ZFN

Question 11

characteristics of gene editing drugs

Answer 11

engineered enzymes
time consuming
low success rate