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Fall 2024 > PHRM 866 Exam 1 > Flashcards

PHRM 866 Exam 1 Flashcards

1
Q

Biotechnology definition

A

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”

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2
Q

Advanced parenterals definition

A

Drugs given by routes other than the digestive tract

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3
Q

What are the 6 Bloom’s Taxonomy levels?

A

Create
Evaluate
Analyze
Apply
Understand
Remember

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4
Q

What are the types of biotech products?

A

Peptides
Proteins
Gene Therapy
Cell Therapy
Vaccines

(Nucleic Acids)

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5
Q

What are 3 examples of gene therapy?

A

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

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6
Q

What are ASOs?

A

Antisense oligonucleotides

Single-stranded nucleic acid

Single-stranded DNA complementary to RNA

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7
Q

What are aptamers?

A

Single-stranded DNA or RNA

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8
Q

What are RNAi?

A

RNA interference
double stranded

double-stranded RNA (dsRNA)

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9
Q

What are mRNA therapeutics?

A

Ex: COVID vaccine
double-stranded RNA (dsRNA)

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10
Q

What is CRISPR?

A

single stranded DNA

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11
Q

What are mAbs?

A

Proteins

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12
Q

What are cell therapies?

A

Whole cells

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13
Q

What are in vivo gene therapies?

A

single-stranded DNA

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14
Q

What are ex vivo gene therapies?

A

DNA

Typically outside of the body “ex vivo”

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15
Q

What are oncolytic viruses?

A

Virus carrying something like DNA
DNA

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16
Q

What are peptides?

A

Tiny fragments in the protein family

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17
Q

What are DNA therapeutics?

A

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

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18
Q

What are protein therapeutics?

A

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

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19
Q

What are cell-based therapies?

A

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

-Used in cancer treatment

20
Q

What are RNA therapeutics?

A

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

21
Q

Is the COVID vaccine a gene therapy?

A

NO

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

*For a gene therapy you must inject DNA

22
Q

Antibodies are what type of therapy?

A

Protein therapy

23
Q

What is an Advanced Therapy Medicinal Product?

A

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

24
Q

What is the aim of regenerative medicine therapy?

A

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

(includes skin patches)

25
Q

What is a NDA?

A

New Drug Approval

for small molecule therapeutics

26
Q

What is a BLA?

A

Biologics License Application

-must be filed for approval of new biologics

27
Q

What does recombinant mean?

A

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

28
Q

What is cloning?

A

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

29
Q

What is the hierarchy of genetic structures?

A

Chromosome -> DNA -> Gene -> Allele

30
Q

What are the features of DNA to know?

A

Double-stranded

Slightly more stable than RNA (no hydroxyl on pentose ring in ribose)

Lives in nucleus

Daughter cell carries DNA of mother cell

31
Q

What are the features of RNA to know?

A

Single-stranded

Not stable

Created from DNA but gets shuffled

Is the building block of proteins

More resistant to UV damage

32
Q

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

A

Splicing
5’ Capping
Polyadenylation

33
Q

What are the 3 locations of biotherapeutics?

A

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

34
Q

What are the therapeutic functions of proteins?

A

-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

35
Q

**What are the types of protein therapeutics?

A

-Receptors
-Antibodies
-Antigens
-Enzymes
-Hormones

36
Q

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

A

Cloning
Expression
Translation

37
Q

What is the purpose of cloning?

A

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

38
Q

What is transfection?

A

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)

39
Q

What is transduction?

A

A way to promote gene internalization that uses viruses

*Can be permanent because the virus can get to the nucleus of the cell

40
Q

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

A

HEK293

41
Q

What are examples of protein and antibody therapeutics vectors?

A

E.coli
yeast
CHO

42
Q

What is glycosylation?

A

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

43
Q

What is a PTM?

A

Post-translational modification

-alter protein function

44
Q

What is protein similarity?

A

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

45
Q

What is homology?

A

A measure of the similarity between the genes coding for two proteins

A high degree of similarity is generally associated with homology

46
Q
A

Fall 2024 (10 decks)

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