Gene Therapy Primer
*This microcourse includes optional CPE Credits and is powered by Biotech Primer, a trusted leader in life science training that simplifies complex topics for non-scientists.
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Optional CPE Credits: 2.0
Overview:
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.
- 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.
By the end of the course, participants will be able to:
- Command the scientific principles and regulatory intricacies behind gene therapy technologies.
- 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.
- Analyze the key factors determining the diseases’ suitability for gene therapy intervention.
- Critique proven and new approaches to offset gene therapy adverse reactions in the clinic.
- Evaluate the FDA approval process and the rigorous standards employed to assess the safety and effectiveness of gene therapies.
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