GMS 6551 Final Exam UPDATED Study
Guide QUESTIONS AND CORRECT
ANSWERS
What is translational research? - CORRECT ANSWERS Translational
research is the process of applying knowledge from basic science research to
develop new therapies or diagnostic tools for human diseases. [PDF
introduction]
What are the four phases of translational research? - CORRECT ANSWERS
T0 (basic research), T1 (pre-clinical research), T2 (clinical research), and
T3 (implementation research).
What is the goal of T0 research? - CORRECT ANSWERS To generate
new knowledge about the underlying mechanisms of disease.
What is the goal of T1 research? - CORRECT ANSWERS To identify
and optimize therapeutic modalities and pre-clinical approaches for treating
human conditions or diseases.
What is the goal of T2 research? - CORRECT ANSWERS To evaluate
the safety and efficacy of new therapies or diagnostic tools in human subjects.
What is the goal of T3 research? - CORRECT ANSWERS To implement
new therapies or diagnostic tools into clinical practice and evaluate their
effectiveness in real-world settings.
What are some examples of therapeutic modalities? - CORRECT ANSWERS
Small molecules, biologics, gene therapies, and cell therapies.
,What are some examples of pre-clinical approaches? - CORRECT ANSWERS
Animal models, in vitro assays, and computational modeling.
What is the purpose of animal models in pre-clinical research? - CORRECT
ANSWERS To evaluate the safety and efficacy of new therapies or
diagnostic tools before testing them in human subjects.
What are some of the advantages of using animal models in pre-clinical
research? - CORRECT ANSWERS The ability to control experimental
conditions, the ability to study complex biological systems, and the ability to
evaluate the safety and efficacy of new therapies or diagnostic tools in a living
organism.
What are the three Rs of animal research? - CORRECT ANSWERS
Reduce, Refine, and Replace.
What are some examples of non-animal approaches that can be used to replace
animal models in pre-clinical research? - CORRECT ANSWERS Cell,
biochemical, and/or in silico simulations.
What are some of the key components of experimental design in pre-clinical
research? - CORRECT ANSWERS Power analysis, randomization,
blinding of investigators, reagent/assay validation, positive/negative controls,
and inclusion/exclusion of data.
What is the difference between accuracy and precision in diagnostic testing? -
CORRECT ANSWERS Accuracy refers to how close a measured value
is to a standard or known value, while precision refers to the degree to which
several measurements provide answers very close to each other.
What are some of the regulatory requirements that impact the movement of pre-
clinical studies into human trials? - CORRECT ANSWERS Safety and
,efficacy testing, ethical considerations, and compliance with Good Laboratory
Practices (GLP) and Good Manufacturing Practices (GMP).
What is the difference between small molecules and biologics? - CORRECT
ANSWERS Small molecules are typically low molecular weight
compounds that can be synthesized chemically, while biologics are typically
large, complex molecules that are produced using living cells or organisms.
What are some examples of diagnostic tools? - CORRECT ANSWERS
Imaging techniques (such as X-rays or MRI), blood tests, and genetic
tests.
What is the purpose of rigor and reproducibility in research? - CORRECT
ANSWERS To ensure that experimental results are accurate, reliable,
and can be replicated by other researchers.
What is a power analysis in experimental design? - CORRECT ANSWERS
A statistical method used to determine the sample size needed to detect a
significant effect in an experiment.
What is blinding in experimental design? - CORRECT ANSWERS A
technique used to prevent bias in experimental design by keeping the
researchers or participants unaware of which group they are in (e.g. treatment
group or control group).
What is the difference between positive and negative controls in experimental
design? - CORRECT ANSWERS A positive control is an experimental
condition that is known to produce a specific result, while a negative control is
an experimental condition that is expected to produce no result. Both types of
controls are used to validate experimental results.
, What is Good Laboratory Practice (GLP)? - CORRECT ANSWERS A
set of guidelines for conducting pre-clinical studies that ensure the quality,
integrity, and reliability of experimental data.
What is Good Manufacturing Practice (GMP)? - CORRECT ANSWERS
A set of guidelines for manufacturing and quality control of drugs,
biologics, and medical devices that ensure their safety, efficacy, and consistency.
What is the role of ethics in translational research? - CORRECT ANSWERS
Plays a critical role in translational research by ensuring that research is
conducted in a manner that is respectful of human subjects, protects their rights
and welfare, and promotes the public good.
What is the purpose of in vitro assays in pre-clinical research? - CORRECT
ANSWERS To evaluate the safety and efficacy of new therapies or
diagnostic tools using cells or tissues in a laboratory setting.
What are some of the advantages of using in vitro assays in pre-clinical
research? - CORRECT ANSWERS The ability to study specific cellular
or molecular processes, the ability to screen large numbers of compounds or
samples, and the ability to reduce the use of animal models.
What is the purpose of computational modeling in pre-clinical research? -
CORRECT ANSWERS To simulate biological processes or drug
interactions using mathematical or computer-based models.
What is the purpose of clinical trials? - CORRECT ANSWERS To
evaluate the safety and efficacy of new therapies or diagnostic tools in human
subjects.
Guide QUESTIONS AND CORRECT
ANSWERS
What is translational research? - CORRECT ANSWERS Translational
research is the process of applying knowledge from basic science research to
develop new therapies or diagnostic tools for human diseases. [PDF
introduction]
What are the four phases of translational research? - CORRECT ANSWERS
T0 (basic research), T1 (pre-clinical research), T2 (clinical research), and
T3 (implementation research).
What is the goal of T0 research? - CORRECT ANSWERS To generate
new knowledge about the underlying mechanisms of disease.
What is the goal of T1 research? - CORRECT ANSWERS To identify
and optimize therapeutic modalities and pre-clinical approaches for treating
human conditions or diseases.
What is the goal of T2 research? - CORRECT ANSWERS To evaluate
the safety and efficacy of new therapies or diagnostic tools in human subjects.
What is the goal of T3 research? - CORRECT ANSWERS To implement
new therapies or diagnostic tools into clinical practice and evaluate their
effectiveness in real-world settings.
What are some examples of therapeutic modalities? - CORRECT ANSWERS
Small molecules, biologics, gene therapies, and cell therapies.
,What are some examples of pre-clinical approaches? - CORRECT ANSWERS
Animal models, in vitro assays, and computational modeling.
What is the purpose of animal models in pre-clinical research? - CORRECT
ANSWERS To evaluate the safety and efficacy of new therapies or
diagnostic tools before testing them in human subjects.
What are some of the advantages of using animal models in pre-clinical
research? - CORRECT ANSWERS The ability to control experimental
conditions, the ability to study complex biological systems, and the ability to
evaluate the safety and efficacy of new therapies or diagnostic tools in a living
organism.
What are the three Rs of animal research? - CORRECT ANSWERS
Reduce, Refine, and Replace.
What are some examples of non-animal approaches that can be used to replace
animal models in pre-clinical research? - CORRECT ANSWERS Cell,
biochemical, and/or in silico simulations.
What are some of the key components of experimental design in pre-clinical
research? - CORRECT ANSWERS Power analysis, randomization,
blinding of investigators, reagent/assay validation, positive/negative controls,
and inclusion/exclusion of data.
What is the difference between accuracy and precision in diagnostic testing? -
CORRECT ANSWERS Accuracy refers to how close a measured value
is to a standard or known value, while precision refers to the degree to which
several measurements provide answers very close to each other.
What are some of the regulatory requirements that impact the movement of pre-
clinical studies into human trials? - CORRECT ANSWERS Safety and
,efficacy testing, ethical considerations, and compliance with Good Laboratory
Practices (GLP) and Good Manufacturing Practices (GMP).
What is the difference between small molecules and biologics? - CORRECT
ANSWERS Small molecules are typically low molecular weight
compounds that can be synthesized chemically, while biologics are typically
large, complex molecules that are produced using living cells or organisms.
What are some examples of diagnostic tools? - CORRECT ANSWERS
Imaging techniques (such as X-rays or MRI), blood tests, and genetic
tests.
What is the purpose of rigor and reproducibility in research? - CORRECT
ANSWERS To ensure that experimental results are accurate, reliable,
and can be replicated by other researchers.
What is a power analysis in experimental design? - CORRECT ANSWERS
A statistical method used to determine the sample size needed to detect a
significant effect in an experiment.
What is blinding in experimental design? - CORRECT ANSWERS A
technique used to prevent bias in experimental design by keeping the
researchers or participants unaware of which group they are in (e.g. treatment
group or control group).
What is the difference between positive and negative controls in experimental
design? - CORRECT ANSWERS A positive control is an experimental
condition that is known to produce a specific result, while a negative control is
an experimental condition that is expected to produce no result. Both types of
controls are used to validate experimental results.
, What is Good Laboratory Practice (GLP)? - CORRECT ANSWERS A
set of guidelines for conducting pre-clinical studies that ensure the quality,
integrity, and reliability of experimental data.
What is Good Manufacturing Practice (GMP)? - CORRECT ANSWERS
A set of guidelines for manufacturing and quality control of drugs,
biologics, and medical devices that ensure their safety, efficacy, and consistency.
What is the role of ethics in translational research? - CORRECT ANSWERS
Plays a critical role in translational research by ensuring that research is
conducted in a manner that is respectful of human subjects, protects their rights
and welfare, and promotes the public good.
What is the purpose of in vitro assays in pre-clinical research? - CORRECT
ANSWERS To evaluate the safety and efficacy of new therapies or
diagnostic tools using cells or tissues in a laboratory setting.
What are some of the advantages of using in vitro assays in pre-clinical
research? - CORRECT ANSWERS The ability to study specific cellular
or molecular processes, the ability to screen large numbers of compounds or
samples, and the ability to reduce the use of animal models.
What is the purpose of computational modeling in pre-clinical research? -
CORRECT ANSWERS To simulate biological processes or drug
interactions using mathematical or computer-based models.
What is the purpose of clinical trials? - CORRECT ANSWERS To
evaluate the safety and efficacy of new therapies or diagnostic tools in human
subjects.
