Final Exam Questions Study Guide GMS 6551 - Expert-Verified Solutions

Final Exam Questions Study Guide GMS 6551 - Expert-Verified Solutions What is translational research? - answer - 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? - answer - T0 (basic research), T1 (pre-clinical research), T2 (clinical research), and T3 (implementation research). What is the goal of T0 research? - answer - To generate new knowledge about the underlying mechanisms of disease. What is the goal of T1 research? - answer - To identify and optimize therapeutic modalities and pre-clinical approaches for treating human conditions or diseases. What is the goal of T2 research? - answer - To evaluate the safety and efficacy of new therapies or diagnostic tools in human subjects. What is the goal of T3 research? - answer - 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? - answer - Small molecules, biologics, gene therapies, and cell therapies. What are some examples of pre-clinical approaches? - answer - Animal models, in vitro assays, and computational modeling. What is the purpose of animal models in pre-clinical research? - answer - 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? - answer - 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? - answer - Reduce, Refine, and Replace. What are some examples of non-animal approaches that can be used to replace animal models in pre-clinical research? - answer - Cell, biochemical, and/or in silico simulations. What are some of the key components of experimental design in pre-clinical research? - answer - 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? - answer - 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? - answer - 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? - answer - 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? - answer - Imaging techniques (such as X-rays or MRI), blood tests, and genetic tests. What is the purpose of rigor and reproducibility in research? - answer - To ensure that experimental results are accurate, reliable, and can be replicated by other researchers. What is a power analysis in experimental design? - answer - A statistical method used to determine the sample size needed to detect a significant effect in an experiment. What is blinding in experimental design? - answer - 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? - answer - A positive control is an experimental condition that is known to produce a specific r