Stem Cell and Gene Therapy Platforms

Quick Overview

In a presentation at the 2021 FAST Science Summit, Jan A. Nolta discussed the platforms for stem cell and gene therapy. These therapies are actively changing healthcare and have the potential to treat a wide range of diseases. Nolta highlighted the importance of specialist facilities and teamwork in manufacturing and delivering these living medicines. She also emphasized the need for natural history studies and understanding the diseases to make a difference in patient outcomes. Nolta discussed different types of stem cells, including hematopoietic stem cells and pluripotent stem cells, and their potential applications in gene therapy. She mentioned ongoing clinical trials and research at UC Davis, focusing on diseases like Angelman syndrome. Nolta also mentioned the use of mesenchymal stem cells for delivering gene editing molecules. Overall, the presentation highlighted the progress and potential of stem cell and gene therapy platforms in revolutionizing healthcare.


I am delighted to be here at the 2021 FAST Science Summit to discuss the exciting advancements in stem cell and gene therapy platforms. These fields are actively changing healthcare and have the potential to revolutionize the treatment of various diseases. In this talk, I will provide an overview of these platforms, their applications, and the ongoing research at UC Davis.

Living Medicines: Cells and Modified Viruses

Stem cell and gene therapies involve the use of living medicines, such as cells and modified viruses, to treat diseases. These therapies are different from traditional pills or vials on a shelf. They require specialized facilities, teamwork, and expertise to manufacture, formulate, and deliver these living medicines. Additionally, natural history studies and collaboration with researchers, MDs, and funding agencies are crucial to understanding diseases and developing effective treatments.

Stem Cell and Gene Therapy Clinical Trials

At UC Davis, we are at the forefront of stem cell and gene therapy research. Currently, we have 48 ongoing clinical trials and have completed two trials, with over 20 more in the pipeline. These trials cover a wide range of diseases and conditions, including Angelman syndrome, diabetes, and spina bifida. Our state-of-the-art Good Manufacturing Practice (GMP) facility allows us to manufacture and deliver cell and gene therapy products for these trials.

Hematopoietic Stem Cell Therapy for Angelman Syndrome

One of our primary focuses is on Angelman syndrome, a neurodevelopmental disorder. We are developing hematopoietic stem cell gene therapy for this condition. Hematopoietic stem cells, which are found in the bone marrow, have the potential to differentiate into various blood cells and microglia, which play a crucial role in the central nervous system. By modifying these stem cells with the correct gene, we aim to restore normal function and improve the symptoms of Angelman syndrome.

Pluripotent Stem Cells and Mesenchymal Stem Cells

In addition to hematopoietic stem cells, we also work with pluripotent stem cells and mesenchymal stem cells. Pluripotent stem cells, including embryonic stem cells and induced pluripotent stem cells (iPSCs), have the ability to differentiate into any tissue type. We use iPSCs to study diseases in a dish, perform drug testing, and develop gene therapy and gene editing techniques. Mesenchymal stem cells, on the other hand, are supportive cells found in the bone marrow. They have a long safety record and can be easily engineered to deliver gene editing tools or other therapeutic molecules to target cells.

Clinical Trials and Future Directions

Our research involves a combination of clinical trials, preclinical studies, and in vitro models to develop and refine stem cell and gene therapy approaches. We are constantly working towards improving the safety and efficacy of these therapies. Our ultimate goal is to provide effective treatments for a wide range of diseases and injuries.


Stem cell and gene therapies have the potential to revolutionize healthcare by providing targeted and personalized treatments for various diseases. The ongoing research at UC Davis and other institutions is paving the way for the development of these therapies. Collaboration between researchers, healthcare providers, funding agencies, and patients is crucial to advancing these fields and improving patient outcomes.

Talk details

  • Title: Stem Cell and Gene Therapy Platforms
  • Author(s): Jan A. Nolta
  • Author(s)’ affiliation: University of California, Davis
  • Publication date: 2022-01-04
  • Collection: 2021 FAST Science Summit