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Gene Therapy Primer presents the scientific principles and regulatory intricacies of manipulating DNA to cure rare diseases. This class uncovers the mechanics of viral vectors and the thought process behind selecting the perfect vector for each unique application. It provides valuable insight into critical factors determining which diseases are suitable for gene therapy intervention and why. Get an in-depth understanding of the FDA approval process and the rigorous standards used to evaluate the safety and effectiveness of gene therapies. Enroll now in Gene Therapy Primer and become well-versed in this medical revolution!

Section 1: Introduction to Gene Therapy

Explore cell biology, genetic disorders, and gene therapy. Understand how gene therapy works in various medical fields, including SMA with Zolgensma as an example.

Section 2: Viral Vector Delivery Options

Dive into gene transfer and tissue specificity in gene therapy. Learn about delivery methods and various viral vectors, including AAV, adenovirus, lentivirus, retrovirus, vaccinia, and MV. Understand the factors influencing vector choice, safety, immunogenicity, and dosage.

Section 3: The Gene Therapy Industry

Explore the FDA’s regulatory landscape in gene therapy, focusing on safety and efficacy measures. Analyze Zolgensma’s approval metrics and delve into the gene therapy drug development process. Discover novel disease targets and survey gene therapy companies.

Five Takeaways


  1. Command the scientific principles and regulatory intricacies behind gene therapy technologies.
  2. Gain proficiency in the mechanics of viral vectors, including the amounts of genetic material each vector type delivers and the cell types of each target.
  3. Analyze the key factors determining the diseases’ suitability for gene therapy intervention.
  4. Critique proven and new approaches to offset gene therapy adverse reactions in the clinic.
  5. Evaluate the FDA approval process and the rigorous standards employed to assess the safety and effectiveness of gene therapies.

Ready to get started?

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  • Closed Captioning in English, Spanish, French, French Canadian, Hindi, Japanese, Korean, Mandarin, Russian, Standard Arabic
  • Transcripts in English, Spanish, French, French Canadian, Hindi, Japanese, Korean, Mandarin, Russian, Standard Arabic
  • Course materials
  • Real-time tracking
  • Progress reporting
  • Accessibility features
  • Corporate account upgrades
  • LinkedIn certificate attachment
  • Verified certificates


  • How long do I have to complete an on-demand course?  You have one year from the date of purchase to complete the course. During that year, you may watch the class an unlimited number of times.
  • What happens if I don’t complete the course in one year?  There are no time extensions. If you want to complete the course, you must purchase it again.
  • How long are the on-demand classes?  Our longest course has a running time of 90 minutes. However, most have a running time of 45 minutes.  The running time is listed for each class on its registration page. The assessment at the end of each course takes between 10-20 minutes to complete.
  • Are there any downloadable class materials? No. All course slides and transcripts remain on your Biotech Primer dashboard, but they are always available for review.
  • What is the format of the on-demand classes?  The online class begins with the class objectives and the course itself and ends with a summary of what you learned. The course is a series of images and text accompanied by a professional voice-over and subtitles. Subtitles are available in 9 languages. The course ends with a quiz to assess your understanding.
  • Can I repeat the course?  You can repeat the course as often as you like within your one-year access.
  • How many times can I take the course quiz?  You have three chances to pass the quiz. A passing score is 75% or higher. You must pass the quiz to receive the class certificate. Your quiz score will be shown on your class certificate.
  • May I share a course I purchased with others?  Each course is to be taken by one individual. If you want to learn as a group, please contact us at for group pricing.
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