PHRM 866 Exam 1 Flashcards
Biotechnology definition
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”
Advanced parenterals definition
Drugs given by routes other than the digestive tract
What are the 6 Bloom’s Taxonomy levels?
Create
Evaluate
Analyze
Apply
Understand
Remember
What are the types of biotech products?
Peptides
Proteins
Gene Therapy
Cell Therapy
Vaccines
(Nucleic Acids)
What are 3 examples of gene therapy?
Cell-based therapy
CAR-T therapy
In vivo gene therapy
What are ASOs?
Antisense oligonucleotides
Single-stranded nucleic acid
Single-stranded DNA complementary to RNA
What are aptamers?
Single-stranded DNA or RNA
What are RNAi?
RNA interference
double stranded
double-stranded RNA (dsRNA)
What are mRNA therapeutics?
Ex: COVID vaccine
double-stranded RNA (dsRNA)
What is CRISPR?
single stranded DNA
What are mAbs?
Proteins
What are cell therapies?
Whole cells
What are in vivo gene therapies?
single-stranded DNA
What are ex vivo gene therapies?
DNA
Typically outside of the body “ex vivo”
What are oncolytic viruses?
Virus carrying something like DNA
DNA
What are peptides?
Tiny fragments in the protein family
What are DNA therapeutics?
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
What are protein therapeutics?
-Inside or outside the cell
-Proteins can go inside the cell but will never go to the nucleus
-Eventually get spit out
What are cell-based therapies?
-Patients get an IV or an entire cell that replaces the patient’s sick cells
-Used in cancer treatment
What are RNA therapeutics?
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
Is the COVID vaccine a gene therapy?
NO
-It does not change the genome (mRNA does not get to the nucleus)
*For a gene therapy you must inject DNA
Antibodies are what type of therapy?
Protein therapy
What is an Advanced Therapy Medicinal Product?
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
What is the aim of regenerative medicine therapy?
To replace, engineer, or regenerate human cells, tissues, or organs to restore or establish normal function
(includes skin patches)
What is a NDA?
New Drug Approval
for small molecule therapeutics
What is a BLA?
Biologics License Application
-must be filed for approval of new biologics
What does recombinant mean?
Engineering cells to carry foreign DNA and become “factories” of therapeutic proteins
What is cloning?
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
What is the hierarchy of genetic structures?
Chromosome -> DNA -> Gene -> Allele
What are the features of DNA to know?
Double-stranded
Slightly more stable than RNA (no hydroxyl on pentose ring in ribose)
Lives in nucleus
Daughter cell carries DNA of mother cell
What are the features of RNA to know?
Single-stranded
Not stable
Created from DNA but gets shuffled
Is the building block of proteins
More resistant to UV damage
What are the 3 steps in the processing of pre-mRNA?
Splicing
5’ Capping
Polyadenylation
What are the 3 locations of biotherapeutics?
Inside the genome (DNA/Gene therapy)
Outside the genome (RNA)
Outside the cell (Protein)
What are the therapeutic functions of proteins?
-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
**What are the types of protein therapeutics?
-Receptors
-Antibodies
-Antigens
-Enzymes
-Hormones
What are the steps to engineering a cell to make a protein?
Cloning
Expression
Translation
What is the purpose of cloning?
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
What is transfection?
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)
What is transduction?
A way to promote gene internalization that uses viruses
*Can be permanent because the virus can get to the nucleus of the cell
What is an example of a viral vector for gene therapy?
HEK293
What are examples of protein and antibody therapeutics vectors?
E.coli
yeast
CHO
What is glycosylation?
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
What is a PTM?
Post-translational modification
-alter protein function
What is protein similarity?
A quantitative measure of the % to which the amino acid sequences of two proteins are identical
What is homology?
A measure of the similarity between the genes coding for two proteins
A high degree of similarity is generally associated with homology
