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Upcoming Event:

Humanized Mice Journal Club

December 13, 2022, 10:30 AM ET (New York)
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Leveraging Gene Therapy to Achieve Long-Term Continuous or Controllable Expression of Biotherapeutics
Chief of the Division of Pediatric Hematology/Oncology/Blood and Marrow Transplant
Nationwide Children’s Hospital

Join us for our next Humanized Mice Journal Club with guest speaker Timothy P. Cripe, MD, Ph.D. The Humanized Mice Journal Club is a monthly opportunity for fellow scientists to present their published work to peers and encourage discussion. The presentations span a variety of preclinical research using humanized mice.

Presentation Abstract

T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been breakthrough technologies; however, CAR-T cells require individualized manufacturing and bispecifics generally require continuous infusions. We created an off-the-shelf, single-dose solution for achieving prolonged systemic serum levels of protein immunotherapeutics via adeno-associated virus (AAV) gene transfer. We demonstrate proof of principle in a CD19+ lymphoma xenograft model using a single intravenous dose of AAV expressing a secreted version of blinatumomab, which could serve as a universal alternative for CD19 CAR-T cell therapy. In addition, we created an inducible version using an exon skipping strategy and achieved repeated, on-demand expression up to at least 36 weeks after AAV injection. Our system could be considered for short-term and/or repeated expression of other transgenes of interest for noncancer applications.

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Leveraging Gene Therapy to Achieve Long-Term Continuous or Controllable Expression of Biotherapeutics
Chief of the Division of Pediatric Hematology/Oncology/Blood and Marrow Transplant
Nationwide Children’s Hospital
Leveraging Gene Therapy to Achieve Long-Term Continuous or Controllable Expression of Biotherapeutics
Chief of the Division of Pediatric Hematology/Oncology/Blood and Marrow Transplant
Nationwide Children’s Hospital
1.800.422.6423 (USA) | 1.207.288.5825 (International)
1.800.422.6423 (USA)
1.207.288.5825 (International)