Gene Therapy: An Overview of Advances and Applications of Gene Therapies

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

Wrap-up

• The Future of Gene Therapy

This course builds on foundational science by exploring complex concepts through engaging biomedical visualizations, clinical applications, and real-world scenarios. To fully benefit from the material, we recommend prior knowledge of basic biology and genetics. This is an advanced course; for those who require more background in genetics, we offer Genetics: Essentials. 

This advanced course surveys current and emerging gene therapies, including in vivo and ex vivo approaches. You’ll explore vectors, gene augmentation strategies, gene editing, and real‑world applications for conditions such as blood disorders, neurological diseases, and inherited retinal disorders.

Highlights include detailed explanations of ex vivo vs. in vivo approaches, vector selection and design, and clinical case examples that illustrate how gene therapies are developed and implemented. The course emphasizes how multidisciplinary advances are transforming patient care.

This course is ideal for clinicians, biomedical researchers, and industry professionals in pharma, biotech, and medical affairs who work with or are preparing to work with gene therapies.

This course is right for you if you work with genetic diseases, advanced therapeutics, or gene‑based technologies—or plan to transition into these areas—and need a structured, clinically grounded understanding of how gene therapies are designed, tested, and used. It’s particularly valuable if you interpret gene therapy data, communicate with patients or customers about these products, or help shape development strategies.

Typical participants include physicians (especially in hematology, neurology, ophthalmology, and pediatrics), fellows, genetic counselors, PhD‑level scientists, regulatory and medical affairs professionals, and R&D and program leaders from gene therapy and rare disease companies.

To benefit fully, you should have prior knowledge of basic biology and genetics, and familiarity with concepts in molecular biology and pharmacology. Foundational genetics courses such as Genetics: Essentials or Genetics Fundamentals are helpful preparation.

You’ll gain a clearer understanding of how gene therapies work, their risks and benefits, and their development pathways, enabling you to engage more effectively in clinical care, trial design, regulatory discussions, and product strategy. This can support advancement into specialized gene therapy roles and enhance your credibility in this highly technical field, supported by a Harvard Medical School certificate.

Gene therapy expertise often involves advanced clinical or scientific degrees plus specialized training in genetics, molecular medicine, or related disciplines, along with hands‑on experience in gene therapy research, clinical trials, or regulatory review. This course provides a structured overview that can accelerate your learning curve, prepare you for more advanced training, and help you decide how deeply to specialize in gene therapy.

Gene Therapy is a 10‑week, online, instructor‑paced course requiring about 1–2 hours per week (15–20 hours total), plus a final exam at the end of the course.

Your progress throughout the course will determine your eligibility for a Certificate of Completion from Harvard Medical School. Earning a certificate requires significant effort; learners must complete all course material by the course end date in order to qualify.

You can bundle up to five courses. Bundle discounts apply to all courses selected. A bundle can consist of courses in the same upcoming enrollment period or the following enrollment period. Bundle-eligible courses include HMX and HealthXcelerate. 

Two-course bundle: 25% discount 
Three-course bundle: 30% discount 
Four-course bundle: 35% discount
Five-course bundle: 40% discount

View all bundle-eligible programs.

Limited program fee assistance is available for participants who qualify. To apply, please submit a request via the fee assistance request form or email learn@hms.harvard.edu for more information.