HMX Pro Genetics
Nucleic Acid Therapeutics
Learn how nucleic acid therapeutics can be used to treat, prevent, and cure diseases.
Even with the thousands of approved small molecule drugs at our disposal, there remains a lack of effective prevention and treatment for many diseases. Fortunately, a new and exciting drug modality has emerged called nucleic acid therapeutics that has the potential to address current unmet needs.
This class of drugs uses nucleic acids and closely related compounds for a variety of applications, including regulating gene expression, stimulating the immune system, editing the genome, or supplying new genetic information, with the goal of treating or preventing disease. Learning about nucleic acid therapeutics is therefore essential for anyone working in health care and related sectors.
This advanced course offers a unique way for professionals to learn from leading Harvard Medical School faculty and industry leaders about nucleic acid therapeutics and the advances in this field that have the potential to treat, prevent, and even cure genetic diseases.
Course Topics
Overview of Nucleic Acid Therapeutics
- What are Nucleic Acid Therapeutics?
- The Promise of Nucleic Acid Therapeutics
- Nucleic Acid Immunity and Delivery Challenges
Removing or Modifying Endogenous Information
- Removing Endogenous Information
- ASO Gapmer Therapeutics
- siRNA Therapeutics
- Modifying Endogenous Information
- ASO Steric Blockers for Exon Inclusion
- ASO Steric Blockers for Exon Exclusion
- Clinical Linkage: Treating Duchenne Muscular Dystrophy with ASOs
Supplying New Information
- Introduction to mRNA Medicines
- Interactive: Developing mRNA Medicines
- mRNA Challenges
- In Focus: What is a Vector?
- mRNA Vaccines
- mRNA Therapeutic Vaccines
- mRNA Therapeutics
- Immunostimulatory Nucleic Acids
- Deep Dive: The Immunostimulatory Effects of mRNA Medicines
- Clinical Linkage: Nucleic Acid-Based Immunotherapy for Melanoma
Permanent Genetic Modifications
- Introduction to Gene Editing
- Gene Editing Considerations
- Deletions with Nucleases
- Templated Modifications
- Base Editing
- Prime Editing
- Clinical Linkage: Treating Sickle Cell Disease with Nucleic Acid Therapeutics
Wrap-up
- The Future of Nucleic Acid Therapeutics
Course Instructors
Caity Anderson, PhD
Lecturer in Genetics, Harvard Medical School
Genetics Curriculum Lead, HMX
Ben Kleinstiver, PhD
Assistant Professor, Harvard Medical School
Art Krieg, MD
Professor, RNA Therapeutics Institute, UMass Chan Medical School
Former CSO, Checkmate Pharmaceuticals, Inc.
Melissa Moore, PhD
Professor, RNA Therapeutics Institute, UMass Chan Medical School
Former CSO, Moderna, Inc.
More Information
Frequently asked questions
Who should take this course?
This course is appropriate for professionals in health care and life sciences whose work relates to nucleic acid therapeutics.
What do participants need to know to succeed in this course?
We recommend knowledge of basic chemistry, biology, and physics, as well as knowledge of key genetics concepts. This is an advanced course; for those who require more background in genetics, we offer HMX Pro Genetics – Essentials.
Not sure which course to take? Gauge your knowledge of genetics by taking this short quiz.
What is the time commitment?
Most people can expect to spend around 15–20 hours total, but this depends on your baseline knowledge, how carefully you take notes, and how seriously you take the assessments. Lessons from the courses are released weekly and remain available until the courses end, so you can work at your own pace. There is a final exam at the end of each course that can be taken any time during the final exam period.
Can participants earn certificates from Harvard Medical School?
There are two certification levels, based on participant scores:
- Certificate of Achievement
- Certificate of Completion
How is this course different from the HMX Gene Therapy course?
In the field of genetics, there are several terms used to describe medicines that are composed of nucleic acids or that target genetic information. At HMX, we’ve chosen to cover this material in two separate courses.
In the HMX Gene Therapy course, the focus is primarily on medicines that act by adding genes either in vivo and ex vivo using viral or non-viral vectors. It provides information about the delivery of these interventions, as well as an introduction to other types of nucleic acid therapeutics, including gene editing. In contrast, the HMX Nucleic Acid Therapeutics course covers mRNA-based medicines, siRNAs, antisense oligonucleotides and immunostimulatory nucleic acids, and explores gene editing techniques in greater depth.
While there is some minor overlap in topics, each course has a unique emphasis and teaches different concepts to allow you to achieve a strong understanding of this exciting field of medicine.