Gene Therapy: An Overview of Advances and Applications of Gene Therapies
- HMX
Group Enrollment Option A program that is a popular choice for organizations and institutions to purchase in bulk.
Explore recent advances in gene therapy and learn about the implications for patient care.
- Online; Instructor-Paced
Lessons are released weekly and remain available until the course ends.
$1,025
Enroll early to receive 10% off. Multi-course bundle pricing is also available.
Early Registration Deadline:
Certificate
There are two certification levels, based on participant scores: Certificate of Achievement; Certificate of Completion
10 Weeks, 1-2 Hours/Week
Most people can expect to spend around 15–20 hours total, but this depends on your baseline knowledge and how carefully you take notes.
On This Page
Overview
In recent years, close collaboration between multiple scientific disciplines has rendered gene therapy a viable treatment option for some genetic conditions, and a promising pursuit for many others such as various cancers, blood disorders, neurological diseases, and inherited retinal disorders. Advancements in vector science, gene editing technologies, and understanding of the molecular underpinnings of disease continue to pave the way for the development of additional gene therapy treatments aimed at improving patient quality of life and prolonging lifespan.
In this advanced HMX course, you will gain an overview of the latest advancements in gene therapy and their transformative impact on patient care. Explore both in vivo and ex vivo gene augmentation strategies, as well as the types of vectors used in different approaches. You will build a strong understanding of the real-world applications of these groundbreaking therapies, including the development of gene therapies that are tailored to treat specific genetic disorders—or even just one patient.
Learning Objectives
- Learn the fundamental concepts of gene therapy including the different modalities, critical terminology, and necessary components
- Gain an understanding of the key differences between ex vivo and in vivo gene therapies, including their unique uses, delivery methods, and challenges
- See how ex vivo and in vivo gene therapies can be clinically applied to treat inherited genetic conditions
Participant Types
Clinicians and other medical professionals; researchers in biomedicine and industry sectors; professionals in biotechnology and health care-related fields
About the Course
HMX online courses bring complex material to life through engaging biomedical visualizations, clinical applications, and true-to-life scenarios paired with lectures from Harvard Medical School faculty and leading experts.
Who Should Apply
This advanced course is appropriate for clinicians and other medical professionals seeking a strong understanding of the principles of gene therapy, with practical examples of the impact and applications of these therapies in clinical settings; researchers in biomedicine and industry sectors looking for comprehensive teaching on gene therapy, with particular focus on delivery and vector design; professionals in biotechnology and health care-related fields who have an understanding of the fundamentals of genetics and would like to learn more about the relevance of gene therapy and its clinical applications.
Course Format
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 toward the beginning of the course session 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 at any time during the multi-week final exam period. The flexible course format makes this an ideal choice for working professionals.
Group Enrollments
HMX courses are ideal for organizations looking to train teams or larger groups. Group pricing is available, making it a cost-effective investment in team development.
Faculty
HMX courses are led by Harvard Medical School faculty, working in collaboration with a multi-disciplinary team of experts in biomedical visualization, assessment, and the science of learning to create a unique learning experience that will stay with you.
Caity Anderson
PhD
- Research Associate in Genetics, Harvard Medical School
- Curriculum Fellow in Online Learning
Connie Cepko
PhD
- Bullard Professor of Genetics and Neuroscience, Harvard Medical School
- Howard Hughes Medical Institute Investigator
- Associate Professor of Pediatrics, Harvard Medical School
- Senior Physician, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
- Medical Director of Clinical Research and Clinical Development, Gene Therapy Program, Boston Children's Hospital
Jason Comander, MD, PhD, Assistant Professor of Ophthalmology, Harvard Medical School / Director of the Inherited Retinal Disorders Service, Massachusetts Eye and Ear
David Corey, PhD, Bertarelli Professor of Translational Medical Science, Harvard Medical School
Florian Eichler, MD, Associate Professor of Neurology, Harvard Medical School / Neurologist and Director of the Leukodystrophy Service, Massachusetts General Hospital
Erica Esrick, MD, Instructor in Pediatrics, Harvard Medical School / Pediatric Hematologist, Boston Children’s Hospital
Rachel Huckfeldt, MD, PhD, Assistant Professor of Ophthalmology, Harvard Medical School / Ophthalmologist, Massachusetts Eye and Ear / Director of the Inherited Retinal Degenerations Fellowship, Massachusetts Eye and Ear
Patricia Musolino, MD, PhD, Assistant Professor of Neurology, Harvard Medical School / Neurologist, Massachusetts General Hospital / Co-Director of the Pediatric Stroke and Cerebrovascular Service, Massachusetts General Hospital / Faculty, Center for Genomic Medicine, Massachusetts General Hospital
David Williams, MD, Leland Fikes Professor of Pediatrics, Harvard Medical School / Chief of the Division of Hematology/Oncology, Boston Children’s Hospital / Associate Chairman, Department of Pediatric Oncology, Dana-Farber Cancer Institute
ADDITIONAL CONTRIBUTORS
Jillian F. Wise, PhD, Instructor in Investigation, Massachusetts General Hospital / Harvard Medical School / Broad Institute
Jordan Said, MD, Internal Medicine Resident, Brigham and Women’s Hospital / Harvard Medical School
Allie Stanton, PhD Candidate, Harvard Medical School
Course Quiz
Are you ready for an advanced course? We recommend knowledge of basic chemistry, biology, and physics, as well as knowledge of key genetics concepts. Gauge your knowledge of genetics by taking this short quiz.
From Our Learners
Hear from HMX students as they share their experiences learning online with Harvard Medical School.
"These are the best online courses I have ever taken by far. Nothing compares to how much knowledge I have gained from these courses."
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