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HMX Pro Genetics
Gene Therapy

Explore recent advances in gene therapy and learn about the implications for patient care.

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. As of today, there are numerous clinical trials underway for gene therapy treatments of various cancers, blood disorders, neurological diseases, and inherited retinal disorders to name a few. Advancements in vector science, gene editing technologies, and our 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. As this field continues to expand, so too will the number of genetic conditions that can be treated and possibly even cured with gene therapy making this an exciting time to delve into this field.

This advanced course offers a unique way for professionals to learn from leading Harvard Medical School faculty about gene therapy and about the advances happening in this field that are ultimately helping to improve the treatment of certain genetic diseases.

Course Topics

Overview of Gene Therapy

  • The History of Gene Therapy
  • The Promise of Gene Therapy

Introduction to Gene Therapy

  • Overview of Gene Therapy
  • Gene Therapy Vectors
  • Vector Considerations
  • Vector Design and Preparation
  • Gene Editing
  • Gene Therapy Challenges
  • Clinical Linkage: Introduction to Inherited Retinal Disorders and Adrenoleukodystrophy

Ex Vivo Gene Therapy

  • Ex Vivo Gene Therapy Indications
  • CAR T Cell Therapy
  • Vectors for Ex Vivo Gene Therapy
  • Conditioning
  • Insertional Oncogenesis
  • Clinical Linkage: Adrenoleukodystrophy

In Vivo Gene Therapy

  • In Vivo Gene Therapy Indications
  • Vectors for In Vivo Gene Therapy
  • Delivery of In Vivo Gene Therapy
  • Overcoming Challenges
  • Clinical Linkage: Inherited Retinal Disorders


  • The Future of Gene Therapy

Download the Gene Therapy course outline

Course Instructors

Caity Anderson

Caity Anderson, PhD

Lecturer in Genetics, Harvard Medical School
Genetics Curriculum Lead, HMX

Connie Cepko

Connie Cepko, PhD

Bullard Professor of Genetics and Neuroscience, Harvard Medical School
Howard Hughes Medical Institute Investigator

Christine Duncan

Christine Duncan, MD

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


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 gene therapy.

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 the fundamentals of genetics. This is an advanced course; for those who require more background in key genetics concepts, we offer HMX Pro Genetics – Essentials.

Not sure if you’re ready for an advanced course? 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 Nucleic Acid Therapeutics 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.