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Master of Advanced Studies in Precision Medicine Therapeutics in Oncology MAS in PMTO
  1. Precision Medicine Therapeutics in Oncology
  2. Curriculum
  3. Cell and Gene Therapy Course

Cell and Gene Therapy

Course Information

PMTO 210 Cell and Gene TherapyDan KaufmanAlysson Muotri

  • 4 units
  • Quarter Offered: Winter
  • Instructors: Alysson R. Muotri, Ph.D. Professor, Dept. of Pediatrics & Cellular Molecular Medicine, UC San Diego School of Medicine | Rady Children’s Hospital, Sanford Consortium; Dan S. Kaufman, MD, PhD, Professor, Dept. of Medicine, Division of Regenerative Medicine, Director of Cell Therapy program, UC San Diego School of Medicine.

Course Description & Learning Objectives


Cell and gene therapies are versatile medical approaches that are becoming increasingly popular. CAR-T cells, AAV viral delivery, ASOs, and CRISPR genome editing enzymes are examples of recent advances that are now part of the clinical vocabulary. However, the rapid development of this field and the complexity of these technologies make it difficult to evaluate when the treatments could be beneficial or to decide what patients would qualify. The course is designed to prepare the students with current knowledge of the diverse cell and gene therapy approaches available for precision medicine. Students will be exposed to several situations where cells and gene therapies could (or could not) be used for medical practices, how these protocols evolved over time, the use of different preclinical model systems,and what is the expectation of this technology in the near and long-term future. Finally, students will also learn how to find reliable resources and educational materials and to critically evaluate these treatments around the world.

The focus is on the scientific rationale for the use of diverse cell and gene therapies in precision medicine. We will go beyond the hype and focus on cutting-edge applications while providing the students with examples of useful and failed pre-clinical and clinical trials.

Learning Outcomes & Skills Acquired from the Course

  • Students in this course will learn the most current cell and gene therapies for forward looking clinicians who can bring novel ideas into the clinic.
  • Students will become facilitators and industry leaders on these emerging technologies to advise on clinical trials, make decisions to enter into this space and become future scientists to join cutting-edge research labs.