Non Viral Gene Delivery – Zhou Lab at Yale University

Prof. James Hansen (left) and Prof. Jiangbing Zhou (right).


Interview with Dr. Jianbing Zhou

  • Could tell us about yourself, your background, and how you got to work on NF?
 I am a Professor of Neurosurgery and of Biomedical Engineering at the Yale Medical School, where I combine my training as a biomedical engineer with the privilege of working alongside esteemed physician colleagues. My primary career goal is to bridge the translational gap between basic biomedical sciences and clinical applications. One of my major research focuses has been on the development of non-viral gene delivery approaches for the treatment of genetic diseases. My journey to NF research began in 2018 through a collaboration with Prof. Robert Kesterson, who was previously at the University of Alabama at Birmingham and is now at the Pennington Biomedical Research Center. Building on this experience, I started to work on developing therapies specifically tailored for NF2 treatment after learning about it from my colleague, Dr. Frank Buono, an Associate Research Scientist in Psychiatry at Yale in 2022.
  • Can you explain, in simple terms, how Nanoparticles and antibodies are being used in your pre-clinical research project for gene delivery and immunotherapy in NF2?
Over the past year, our research efforts have centered around identifying effective therapeutic approaches for treating NF2. Through a comprehensive preliminary screening process, we have selected gene replacement therapy as our primary focus. This therapy is delivered using either polymeric nanoparticles or a cell-penetrant antibody. In earlier studies, we successfully developed innovative polymeric nanoparticles capable of efficiently delivering a range of genetic payloads, showing promising potential for treating genetic diseases. However, what most excites us is the antibody approach, which was a collaborative effort with Prof. James Hansen, a distinguished physician scientist at Yale. Prof. Hansen’s expertise lies in developing cell-penetrating antibodies for clinical applications, and our collaborative journey spans over eight years. The antibody approach is particularly intriguing because the specific antibody we developed exhibits two remarkable properties. Firstly, it effectively delivers the NF2 gene into diseased cells. Secondly, it elicits an innate immune response by activating the cGAS–STING signaling pathway. This dual functionality makes the antibody approach highly promising for NF2 treatment.
  • How do you envision this research advancing the current treatment options for individuals with NF2? What potential benefits might it offer to the NF2 community?

The gene replacement approach, whether delivered through nanoparticles or antibodies, has a major advantage over current NF2 treatments as it directly addresses the genetic cause of the disease. The antibody-based therapy may also eliminate distal tumor cells by triggering an immune response. If successful, our approach could offer a novel and effective management strategy for the NF2 community.

  • Are there any specific challenges or risks associated with using antibodies for gene delivery and immunotherapy that the NF2 community should be aware of?

At present, we are not aware of any major challenges or risks associated with using antibodies for gene delivery and immunotherapy. However, we remain vigilant and will closely monitor these aspects in our future studies to ensure the safety and efficacy of our approach.

  • What stage is your research currently at, and what are the next steps in terms of bringing this approach closer to clinical trials or potential implementation for NF2 patients?

We are currently in the process of rigorously characterizing the specific antibody for NF2 treatment, using relevant disease models. If our findings prove promising, our next step will involve collaborating with industrial partners to humanize the antibody and prepare for clinical trials.

  • How can individuals within the NF2 community stay informed about the progress of your research and any future developments related to this project? Is there a way for them to participate or contribute in any manner?

Rest assured that we will keep the NF2 community informed about any significant progress we make. Stay tuned for updates! As we move forward, we may require assistance from the community, especially in the realm of clinical translation, following the successful validation study.

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