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Ben Gurion FTA #1

 

FTA: Bio-inspired Nano-carriers for Sub-Cellular Targeted Therapeutics

Prof. Joseph Kost, FTA leader 

Dr. M. Zohar, coordinator       

Profs. Ashraf Brik, Anne Bernheim, Smadar Cohen, Levi Gheber, Rony Granek, Eyal Nir, Hanna Rapaport,  Assaf Rudich,  Ben-Gurion University

Prof. Ziv Reich, the Weizmann Institute of Science

Prof. Jacob Bar-Tanna, Hebrew University


This FTA program is focused on developing bio-inspired nanocarriers (NCs) aimed at sub-cellular targeting of therapeutics. Uniquely, this program presents a comprehensive  drug delivery approach  that includes aspects such as:  design of new NCs, synthesisa nd characterization, intracellular trafficking, sub-cellular recognition, and localized cargo discharge, up to the level of in-vivo proof of efficacy.

The  ability  of  a  molecule  to  selectively  find  its  target  at  sub-cellular  levels  is  essential  for  safely  exerting  its  therapeutic potential. A drug molecule with no defined specificity for a particular organelle would either require sufficiently long metabolic stability to allow for random interaction with organelle-specific target, or an efficient targeting strategy for the intended sub-cellular compartment. Complementarily, efficient specific sub-cellular targeting can assist in avoiding side-effects that result from the drug acting on off-targets.

Using nanoparticles as drug carriers takes advantage of their unique properties, such as enhanced loading, dense packing, controlled cargo release, stability and importantly -- ease of functionalization for sub-cellular targeting. We aim to develop  tailored nanoparticles, uniquely made of biocompatible materials (peptides, polysaccharides, lipids) for the targeted delivery of therapeutic agents into specific  sub-cellular compartments.

The program implements approaches and novel chemistries to achieve targeting of nano-size drug carriers to specific sub-cellular compartments (cytosol, mitochondria, nucleus), and  addresses the multi-step complexity  from NC  design  to  investigating  mechanisms  of  NC  transport  and  specific  targeting  in  sub-cellular compartments, up to the level of  in-vivo proof of concept of efficiency. To achieve this goal, an interdisciplinary group of experts for each of these essential steps has teamed up.  Our therapeutic molecules range from nucleic acids such as plasmids and siRNA, to chemotherapeutic agents and small molecules for the treatment of metabolic syndrome-related diseases (such as obesity and insulin resistance) and cancer.