Lecture #11

Question 1

RNA interference

Answer 1

antisense oligonucleotides (ASO): reduce mRNA level, this less protein will be made
onapttro - an RNAi therapeutic for people with a rare and deadly genetic disorder called hereditary transthyretin-mediated amyloidosis

Question 2

mRNA medicine

Answer 2

introduce exogenous mRNA into the body, so cells can make proteins based on the introduced mRNA
COVID-19 vaccine

Question 3

Monoclonal antibody and antibody-drug conjugate

Answer 3

type of Y shape protein that specifically binds to its target to block the function of that target protein; or to help to recognize a specific group of cells that express that target protein
PD1 and PD-L1 drugs are monoclonal antibodies

Question 4

Gene therapy

Answer 4

virus as a vehicle to deliver gene
FDA approved adneno-associated virus drug Luxturna to treat a rare form of inferitied vision loss

Question 5

CRISPR-mediated gene editing and mutation repair

Answer 5

trial of an experimental CRISPR-Cas9 therapy for the blood disorder beta-thalassemia has been launched

Question 6

Stem cell (iPSCs) therapy

Answer 6

most are still being developed; has potential to generate any cell types in body to replace damaged tissue

Question 7

Stem cell therapy targets

Answer 7

cell as a whole

Question 8

CRISPR based gene correction therapy targets

Answer 8

DNA

Question 9

mRNA medicine and ASO target

Answer 9

RNA

Question 10

Monoclonal antibodies target

Answer 10

protein

Question 11

Antisense oligonucleotides

Answer 11

block the mRNA directly to reduce the protein being made from that particular mRNA
reduce something rather than introduce something

Question 12

mRNA medicine

Answer 12

scientists generated an mRNA seqeunce that codes for the virus spike protein –> RNA sequence, a blueprint for making the spike, is swathed in a lipid coating for delivery –> cells read info in the mRNA sequence to produce millions of copies of the spike protein –> protein fragments spur the immune system to produce antibodies that can protect when a real virus enters the body
making the exogenous mRNA stable is the key

Question 13

Monoclonal antibodies and ADC

Answer 13

antibody is a type of protein that very specifically binds to its target and often blocks the function of its target
antibodies made by B cell: neutralize and block invaders from entering and infection other cells, also tags them for destruction

Question 14

Gene therapy with adeno-associated virus

Answer 14

transgene packaged into AAV vectors –> one-time IV infusion, AAV’s carrying transgene target the liver –> in the liver, AAV vectors deliver transgene to nucleus of liver cells to enable production of therapeutic protein

Question 15

Zolgensma (AAV)

Answer 15

SMA is a neuromuscular disorder caused by a mutation in SMN1 gene –> muscle atrophy
lacking 1 protine, need to supply one healthy gene
biologic drug consisting of AAV capsids that contains a SMN1 transgene along with synthetic promoters
accumulates in liver
most expensive drug on market

Question 16

CRISPR/Cas9

Answer 16

cuts double strand of DNA into 2 pieces
cell transfected with an enzyme complex containing: guide molecule, healthy DNA molecule, DNA cutting enzyme –> synthetic guide molecule finds the target DNA strand –> enzyme cuts off target DNA strand –> defective DNA strand replaced with healthy copy
all approved uses for invitro

Question 17

Stem cell therapies

Answer 17

still in early stage
not FDA approved
induced pluripotent stem cells are the most promising; also have neural stem cells, embryonic stem cells, and progenitor cells