2. Gene Therapy

Question 1

2 Types of Drugs

Answer 1

1. Small molecules (chemicals)- MOST DRUGS IN THIS CATEGORY
2. Biologics (large biomolecules) ie. proteins, genes, cell therapies, vacancies

Question 2

3 Top gene disease trials

Answer 2

1. cancer
2. genetic disease
3. infection

Question 3

Gene therapy approval 2012

Answer 3

• lipoprotein lipase (rare disorder) deficiency, $1M, 31 patients treated worldwide

Question 4

Gene therapy approval 2017

Answer 4

• for hereditary disease
• CAR-T therapies (cellular therapies involving genetic manipulation)

Question 5

Gene therapy approval 2019

Answer 5

gene therapy for Spinal muscular atrophy (SMA)

Question 6

Today, there are ___ FDA approved gene therapies

Answer 6

5

Question 7

Many receptor proteins are found in the ______ ______

Answer 7

plasma membrane

Question 8

______ Directs the cell’s activities

Answer 8

DNA

Question 9

______ are organization units of DNA

Answer 9

Genes

Question 10

DNA Structure Base Pairs

Answer 10

A-T
C-G

Question 11

DNA

Answer 11

the cell’s genetic information

Question 12

mRNA

Answer 12

the DNA’s messenger

Question 13

Proteins

Answer 13

are huge chains of amino acids & carry out cell functions

Question 14

Codon

Answer 14

every group of 3 bases on an mRNA (ie:UGA)

Question 15

Each ______codes for an amino acid

Answer 15

codon

Question 16

Amino acids

Answer 16

building blocks of proteins

Question 17

RNA is made up of…

Answer 17

AUGC

Question 18

Genetic code

Answer 18

converts RNA information to amino acid information

Question 19

Genetic variation & mutations

Answer 19

An individual’s DNA can be mutated by errors during DNA copying (DNA replication) or by the action of DNA damage

Question 20

All proteins are made out of ____ building blocks, called amino acids

Answer 20

21

Question 21

Hydrophobic amino acids

Answer 21

have carbon-rich side chains, which don’t interact well with water

Question 22

Hydrophilic or polar amino acids

Answer 22

interact well with water

Question 23

Charged Amino Acids

Answer 23

interact with oppositely charged amino acids or other molecules.

Question 24

Building blocks of protein

Answer 24

C, O, H, N, S

Question 25

Building Blocks of amino acids

Answer 25

• carboxyl group
• amino group
• alpha carbon
• side chain

Question 26

Antibodies

Answer 26

protect us from disease by recognizing and binding to pathogens such as viruses, and targeting them for destruction by the immune system

Question 27

Gene therapies

Answer 27

when disease mechanism involves a single gene

Question 28

2 Case Studies for gene therapy

Answer 28

1. RPE65 Gene therapy to the eyes
2. Beta thalassemia therapy (cant produce enough beta globin)