Genetic Underpinnings of Synaptic Disease
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Genetic Underpinnings of Synaptic Disease
This course is part of Synaptic Pharmacology Specialization
Instructor: Anna Lee, Ph.D.
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March 2026
6 assignments
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There are 3 modules in this course
This course explores how genetic variation shapes synaptic function, neural circuits, and behavior. Learners will examine how specific gene mutations alter communication between neurons and how these molecular changes scale up to influence cognition, development, and addiction.
The course begins by investigating genetic disruptions of synaptic proteins using Fragile X syndrome as a central example. Learners will explore how mutations in key regulatory genes impair synaptic plasticity, alter neural development, and contribute to neurological symptoms, while also examining current challenges and emerging therapeutic strategies for genetic brain disorders. The course then introduces genetic knockout models as powerful tools for identifying gene function, highlighting research on nicotinic acetylcholine receptor subunits and human genetic mutations that serve as natural experiments in understanding disease mechanisms. In the final module, learners integrate these concepts to study complex synaptic modulation within the brainβs reward pathway, using nicotine addiction and smoking as case studies. The course explores how multiple neurotransmitter systems interact within reward circuits to drive motivation and reinforcement, and concludes with an overview of smoking cessation therapies, emphasizing the pharmacokinetic and pharmacodynamic principles that determine their clinical effectiveness. This course is designed for learners with a background in biology who are interested in neuroscience, genetics, pharmacology, or addiction science. It is especially well suited for learners preparing for careers in medicine, biomedical research, psychology, pharmacy, or other health sciences.
This module explores how genetic alterations affect synaptic function, using Fragile X syndrome as a key example. We will learn about the genetic mutations that cause Fragile X syndrome, how these changes disrupt synaptic activity, and the current challenges and promising advances in treating genetic forms of neurological disease.
What's included
3 videos2 readings2 assignments
3 videosβ’Total 14 minutes
- Introduction to FXS and epigenetic modificationβ’5 minutes
- Genetic alterations in FXSβ’3 minutes
- Challenges in treatment of genetic diseaseβ’6 minutes
2 readingsβ’Total 40 minutes
- Course Syllabusβ’10 minutes
- Gene therapy: principles, challenges and use in clinical practiceβ’30 minutes
2 assignmentsβ’Total 60 minutes
- Genetic causes of synaptic disease practice quizβ’30 minutes
- Genetic causes of synaptic disease quizβ’30 minutes
This module examines the use of genetic knockout models in identifying the roles of specific genes, highlighting studies of nicotinic acetylcholine receptor subunits as a case example. The accompanying reading discusses human genetic mutations that act as natural experiments, offering insight into gene function and disease mechanisms.
What's included
4 videos1 reading2 assignments
4 videosβ’Total 23 minutes
- Nicotinic acetylcholine receptors in the brainβ’5 minutes
- Nicotinic receptor subtypesβ’5 minutes
- Exploring function with pharmacologyβ’7 minutes
- Exploring function with genetic knockoutsβ’6 minutes
1 readingβ’Total 30 minutes
- Natural human knockouts and the era of genotype to phenotypeβ’30 minutes
2 assignmentsβ’Total 60 minutes
- Determining function with genetic knockouts practice quizβ’30 minutes
- Determining function with genetic knockouts quizβ’30 minutes
In this module, we will bring together concepts from the course to examine complex synaptic modulation using nicotine addiction and smoking as examples. We will introduce the brainβs reward pathway and the key neural circuits involved in motivation and reinforcement. Within each synapse of this simplified reward circuit, we will explore how different neurotransmitters influence overall circuit activity. The module will also include a brief overview of the history of the cigarette epidemic and conclude with a discussion of common drugs used for smoking cessation, examining their pharmacokinetics and pharmacodynamics as key aspects of their efficacy.
What's included
4 videos1 reading2 assignments
4 videosβ’Total 21 minutes
- Introduction to the reward pathwayβ’6 minutes
- Complex synaptic modulation: Example of nicotine addictionβ’7 minutes
- Pharmacotherapy for smoking cessationβ’7 minutes
- Conclusion to Synaptic Pharmacologyβ’1 minute
1 readingβ’Total 10 minutes
- Rise of cigarettes after WW2β’10 minutes
2 assignmentsβ’Total 60 minutes
- Complex synaptic modulation practice quizβ’30 minutes
- Complex synaptic modulation quizβ’30 minutes
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University of Minnesota
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University of Minnesota
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Status: Free TrialCategory: Credit offered - U
University of Minnesota
Course
Status: Free TrialCategory: Credit offered - U
University of Minnesota
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Status: Free TrialCategory: Credit offered
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