Ciclo de Seminarios 2019
2019 8 AGO
Jueves 8 de Agosto
11:00 hs. - Auditorio Emma Pérez Ferreira
Edificio TANDAR
"Turning Academic Discoveries into Novel Therapeutic Modalities"
Dan Peer
(*)
Laboratory of Precision Nanomedicine, Dept. of Cell Research & Immunology, George S. Wise Faculty of life Sciences
Dept. of Material Sciences and Engineering, Faculty of Engineering
The Center for Nanoscience & Nanotechnology
Cancer Biology Research Center, Tel Aviv University, Israel
RESUMEN:
Accumulating work points out relevant genes and signaling pathways hampered in human disorders as potential
candidates for therapeutics. Developing nucleic acid-based tools to manipulate gene expression, such as
siRNAs, mRNA and genome editing strategies, open up opportunities for personalized medicine. Yet, although
major progress was achieved in developing RNA targeted delivery carriers, mainly by utilizing monoclonal
antibodies (mAbs) for targeting, their clinical translation has not occurred. In part because of massive
development and production requirements and high batch-to-batch variability of current technologies, which
relies on chemical conjugation. Here we present a self-assembled modular platform that enables to construct
theoretically unlimited repertoire of RNA targeted carriers. The platform self-assembly is based on a
membrane-anchored lipoprotein, incorporated into RNA-loaded lipid nanoparticles that interact with the
antibody Fc domain. We show that a simple switch of 8 different mAbs, redirects specific uptake of siRNAs by
diverse leukocyte subsets in vivo. The platform therapeutic potential is demonstrated in an inflammatory
bowel disease model, by targeting colon macrophages to reduce inflammatory symptoms, and in Mantle Cell
Lymphoma xenograft model, by targeting cancer cells to induce cell death and improve survival. In addition, I
will discuss novel approach for delivering modified mRNA to specific cell types in vivo utilizing this
platform. This modular delivery platform can serve as a milestone in turning precision medicine feasible.
‡ con el auspicio de Amigos de la Universidad de Tel Aviv en Argentina
* Professor and Director of the Laboratory of Precision NanoMedicine at Tel Aviv University (TAU). Vice Dean for Research at the George S. Wise Faculty of Life Sciences. Chair of TAU Cancer Biology Research Center (the biggest Cancer Center in Israel, including 17 affiliated hospitals). Founding and Managing Director of the SPARK program of Translational Medicine at TAU. He has more than 100 pending and granted patents. Based on his work, five spin-off companies were generated aiming to bring innovative personalized medicine into clinical practice. He received more than 30 awards and honors and he serves on the scientific advisory board of more than 10 companies, and on the editorial board of more than 15 journals. He is an associate Editor of the Journal of Controlled Release. He was president of the Israeli Chapter of the Controlled Release Society, and member of the Board of the Israel Young Academy of Science. Prof. Peer's work was among the first to demonstrate systemic delivery of RNA molecules using targeted nanocarriers to the immune system and he pioneered the use of RNA interference (RNAi) in immune cells. His lab was the first to show systemic, cell specific delivery of modified mRNA to cells to induce therapeutic gene expression of desired proteins within the immune system that has enormous implications in cancer and inflammation.