Midterm (Modules 2-7) Flashcards
What are some differences between small molecules and biologics
biologics are large, unstable, usually injected, and made from living things, resulting sometimes in a heterogeneity
What are the steps of making recombinant DNA
I really like fucking people
Isolate
Restrict and ligage
fermentation
purification
What is the difference between biotechnology and biosciences
biotechnology is creating a product using living things and biology where as biosciences is the study of biology
What form of DNA is patentable
cDNA
What is cDNA
using reverse transcriptase, you can make a complementary stand of DNA that does not have any introns. This is considered not natural
Name 3 mechanisms of therapeutic action for mABs
- alter immune response
- trigger cell death
- disrupt signaling proteins
What are the two requirements from researchers before beginning a clinical trial
review and approval from IRB and file a IND with the FDA
How long is a typical patent for
6-10 years
What is pharmacogenetics
how someones genetics influences how they respond to drugs
what is pharmacokinetics
what the body does to the drug
what is pharmacodynamics
what the drug does to the body, think pharmaco-D what the DRUG DOES
What types of actions could drugs have?
work directly on the receptor, block mechanism of action, stablize action, direct harmful chemical to destroy cell, directly beneficial chemical
What is the largest group of biologics sales
monoclonal antibodies
what are some ways recombinant DNA can be uses
crops and GMOs, therapeutics,
What are some advantages of making proteins
can be produced in large amounts in a short amount of time,
What are some disadvantages of making proteins
multiple proteins can form aggregates, unstable, physical nature of some proteins can change theraputic nature
What is clinical research
research conducted with human subjects for which the investigator directly interacts with the subject
what are the critical issues with a cross over study
- the order in which treatments are administered
- the carry-over between treatments/wash out period
what is the IRB
the guardian of clinical research, reviews, protocols, informed consent, and the risk benefit ratio
what things are exempt from the IRB
Emergency use, anything with taste and food
What needs to be submitted to the FDA?
clinical research investigator, conditions of study, purpose, contact information
What is the process to initiate clinical trials?
- submit investigation of new drug application
- product must have potential benefit and be proven safe in preclinical models
What is -omics
a holistic approach to looking at what is going on in the cells
what is systems biology
the integration of different -omics to understand what is happening
define genomics
a persons genome
define metagenomics
genetic material directly from environment
define pharmacogenomics
study of genetic variation effect on drug response
define pharmacogenomics
define proteomics
define bioinformatics
What 3 groups make up proteins
the r-group, the carb
what form of structure in a protein offers its function
secondary structure
what happens in the primary structure
sequence of amino acids
what happens at the secondary structure
disulfide bonds forming alpha helices and beta sheets
what happens at the tertiary level
three dimensional folding
what happens at the quaternary level
what happens to proteins as you increase temperature or effect pH
Denaturing,
what is a competitive inhibitor
an inhibitor that competes with the substrate and there for slows it down
what is an allosteric inhibitor
and inhibitor that binds to the enzyme and causes a change in shape to the enzyme, making it so the enzyme unable to bind to the substrate
what was the first organism to have a compete genome sequenced?
a bacterium
what was the first genetically modified organism?
a bacterium
what are three important characteristics for a target protein
druggable,
what current problems do we face in drug development
what are some solutions to address current problems with drug development?
what is the SBIR
What is the SBA?
what quality requirements are there in drug discovery?
what are good practices?
what are good manufacturing practices?
what are good laboratory practices?
what is the IHC and its role?
molnupirav
What is genetic engineering
direct manipulation of an organisms genome using biotechnology, also called genetic modification
True or False: Restriction enzymes are not required for the genetic engineering process
false
which organism was first genetically modified?
bacterium
what omics includes structural biology, algorithms, and other disciplines?
bioinformatics
This is used to optimize drug therapy with respect to the patients’ genotype, to ensure maximum efficacy with minimal adverse effects
pharmacogenomics
what is the difference between the term genomics and genetics.
genetics is the science of heredity but genomics is the study of genes, their functions and all related technical aspects
What organizations need to approve a genetically modifies organism for release?
USDA, FDA, and EPA
True or false gene therapy can be applied to somatic but not germline cells
false
What is the meaning of disease pivotal points? what are they important and when do we have to consider them?
these are points involved in the disease pathway and can regulate the disease, manipulation of these points can impact the disease, and we consider them during the discovery phase
In dose escalation protocol, when is toxicity found?
when the max tolerated level is one level below the observed level
what is the function of the IRB
to approve, disapprove, modify, and review biomedical and behavioral research involving humans
what is one criteria for IRB approval?
informed consent and it is appropriately documented
what is the process to initiate clinical trials?
the decision on whether or not to approve a drug or not is based on
benefit vs risk assessment
what are two incidents where IRB approval is exempt
emergency use and things involving food and taste
how are clinical trials initiated
use of human subjects, IRB approval, and cooperation of clinical centers
What characteristics would make a good choice as a target for a protein drug
a cell surface receptor and a cell surface receptor ligand
What type of bond is the nitrogenous base linked to the ribose or deoxyribose sugar
glycosidic bond
What adaptor is key to converting the triplet codons of mRNA into amino acids
tRNA
What are some differences in prokaryotic and eukaroytic cells protein synthesis?
what direction is mRNA translated
greatest to least, 5’ to 3’, start with the greatest
what are 3 types of mutations which may affect DNA
point mutation, insertion, deletion
Grants vs contracts
What is IND
what do you need for IRB approval?
NIH vs NSF
specific steps of transcription
specific steps of translation
what are some advantages of mRNA vaccines
what are disadvantages of mRNA vaccines?
how do mRNA vaccines differ from molnupirva
How do enzymes work
What are protein protein interactions?
What are proteins
large molecules that contain fundamental biological and chemical components of all living organisms
what is the largest family of targeted drugs
G protein coupled receptors
what are 3 characteristics of clinical research
- seek to identify the most effective and most efficient interventions, treatments, and services
- Examine the distribution of disease, factors that affect health, and how people make health-related decisions
- Involves a particulare person or group of people or uses materials from human
What is a crossover study?
administering two or more experimental therapies, one after another, in a specified or random order to the same group
how do omics differ from not traditional studies
not hypothesis driven
Why is cell culture technology important?
research, toxicity testing, gene therapy, genetic engineering, modeling systems
What are some common mammalian cell lines?
approach for genetic engineering of cell line
Identification of genes/proteins that are specifically up-regulated in bioprocessing conditions (-omics approach)
Engineering of cells and selection of a new cell line
Engineering of cells to over-express the gene(s) of interest (historical approach)
Examine the effect and select a new cell line
Further understanding of genes/pathways directly regulated by the gene of interest
Bcl-2 over-expression
Increases cell viability
Prolongs culture duration
Reduces serum dependency
Improves nutrient metabolism
Protects cells in stressful conditions
Enhances adaptation in serum-free media
