PHRM 866 Exam 1

Question 1

Biotechnology definition

Answer 1

The use of advances in molecular biology for applications in human health

“An integrated application of scientific and technical understanding of a biologic process or molecule to develop a useful product”

Question 2

Advanced parenterals definition

Answer 2

Drugs given by routes other than the digestive tract

Question 3

What are the 6 Bloom’s Taxonomy levels?

Answer 3

Create
Evaluate
Analyze
Apply
Understand
Remember

Question 4

What are the types of biotech products?

Answer 4

Peptides
Proteins
Gene Therapy
Cell Therapy
Vaccines

(Nucleic Acids)

Question 5

What are 3 examples of gene therapy?

Answer 5

Cell-based therapy
CAR-T therapy
In vivo gene therapy

Question 6

What are ASOs?

Answer 6

Antisense oligonucleotides

Single-stranded nucleic acid

Single-stranded DNA complementary to RNA

Question 7

What are aptamers?

Answer 7

Single-stranded DNA or RNA

Question 8

What are RNAi?

Answer 8

RNA interference
double stranded

double-stranded RNA (dsRNA)

Question 9

What are mRNA therapeutics?

Answer 9

Ex: COVID vaccine
double-stranded RNA (dsRNA)

Question 10

What is CRISPR?

Answer 10

single stranded DNA

Question 11

What are mAbs?

Answer 11

Proteins

Question 12

What are cell therapies?

Answer 12

Whole cells

Question 13

What are in vivo gene therapies?

Answer 13

single-stranded DNA

Question 14

What are ex vivo gene therapies?

Answer 14

DNA

Typically outside of the body “ex vivo”

Question 15

What are oncolytic viruses?

Answer 15

Virus carrying something like DNA
DNA

Question 16

What are peptides?

Answer 16

Tiny fragments in the protein family

Question 17

What are DNA therapeutics?

Answer 17

Gene therapy!

Patient receives gene therapy, DNA goes into the cell and to the nucleus
When the cell divides the DNA is transferred to all daughter cells
*DNA of cells is changed forever

Question 18

What are protein therapeutics?

Answer 18

-Inside or outside the cell
-Proteins can go inside the cell but will never go to the nucleus
-Eventually get spit out

Question 19

What are cell-based therapies?

Answer 19

-Patients get an IV or an entire cell that replaces the patient’s sick cells

-Used in cancer treatment

Question 20

What are RNA therapeutics?

Answer 20

Example: COVID vaccine

-RNA lives in the cytoplasm and DOES NOT GO TO THE NUCLEUS
-RNA gets changed to proteins in the cytoplasm
-These only last a short time because it can only last as long as there is mRNA present and able to make protein

Question 21

Is the COVID vaccine a gene therapy?

Answer 21

NO

-It does not change the genome (mRNA does not get to the nucleus)

*For a gene therapy you must inject DNA

Question 22

Antibodies are what type of therapy?

Answer 22

Protein therapy

Question 23

What is an Advanced Therapy Medicinal Product?

Answer 23

A medicine for human use that is based on genes, cells, or tissue engineering

(often combined with a medical device)

Includes cell therapy, gene therapy, ATMP, CAR-T, and AAV gene therapy

Question 24

What is the aim of regenerative medicine therapy?

Answer 24

To replace, engineer, or regenerate human cells, tissues, or organs to restore or establish normal function

(includes skin patches)

Question 25

What is a NDA?

Answer 25

New Drug Approval *for small molecule therapeutics*

Question 26

What is a BLA?

Answer 26

Biologics License Application -must be filed for approval of new biologics

Question 27

What does recombinant mean?

Answer 27

Engineering cells to carry foreign DNA and become "factories" of therapeutic proteins

Question 28

What is cloning?

Answer 28

Turning engineered cells into the type of high-quality biotech "factory" required for therapy Expansion of exact DNA inside an organism to create a larger number of copies

Question 29

What is the hierarchy of genetic structures?

Answer 29

Chromosome -> DNA -> Gene -> Allele

Question 30

What are the features of DNA to know?

Answer 30

Double-stranded Slightly more stable than RNA (no hydroxyl on pentose ring in ribose) Lives in nucleus Daughter cell carries DNA of mother cell

Question 31

What are the features of RNA to know?

Answer 31

Single-stranded Not stable Created from DNA but gets shuffled Is the building block of proteins More resistant to UV damage

Question 32

What are the 3 steps in the processing of pre-mRNA?

Answer 32

Splicing 5' Capping Polyadenylation

Question 33

What are the 3 locations of biotherapeutics?

Answer 33

Inside the genome (DNA/Gene therapy) Outside the genome (RNA) Outside the cell (Protein)

Question 34

What are the therapeutic functions of proteins?

Answer 34

-Replace proteins that are deficient or abnormal -Augment an existing pathway -Provide a novel or deficient function or activity -Interfere with a molecule or biological process -Deliver other compounds or proteins

Question 35

**What are the types of protein therapeutics?

Answer 35

-Receptors -Antibodies -Antigens -Enzymes -Hormones

Question 36

What are the steps to engineering a cell to make a protein?

Answer 36

Cloning Expression Translation

Question 37

What is the purpose of cloning?

Answer 37

To make sure that every individual bacterium in a culture has the same desired genes after transfection Generates billions of genetically identical bacteria to serve as factories of protein

Question 38

What is transfection?

Answer 38

A way to promote gene internalization that does not use viral vectors -Ex: heat, LMP's, liposomes, etc -Mostly transient (gene is eventually lost) (most do not make it to the nucleus)

Question 39

What is transduction?

Answer 39

A way to promote gene internalization that uses viruses *Can be permanent because the virus can get to the nucleus of the cell

Question 40

What is an example of a viral vector for gene therapy?

Answer 40

HEK293

Question 41

What are examples of protein and antibody therapeutics vectors?

Answer 41

E.coli yeast CHO

Question 42

What is glycosylation?

Answer 42

Covalent linking of polysaccharide chains to specific sites of the amino acid chain in a protein post-translationally Post-translational modification Creates glycoproteins Can increase the conformational stability of proteins

Question 43

What is a PTM?

Answer 43

Post-translational modification -alter protein function

Question 44

What is protein similarity?

Answer 44

A quantitative measure of the % to which the amino acid sequences of two proteins are identical

Question 45

What is homology?

Answer 45

A measure of the similarity between the genes coding for two proteins A high degree of similarity is generally associated with homology