Principal Investigator: Prof. Niveen Khachab

Poster Presenter: Dana Al Kelabi

Lab: SHMS

Nature-driven crystalline coordination polymer for nucleus targeting CRISPR/CAS9 delivery

 

Abstract

 

The rapid advances in gene therapy over the past few decades have established the potential for such a method to be used as a standard therapeutic option for the treatment of several conditions including cancers, Autoimmune diseases, infectious diseases...etc. The key factor in the success of gene therapy is the correct choice of delivery vector. Two main delivery vector categories have been applied in gene therapy, viral and non-viral vectors. Viral-based vectors are limited by the low loading capacity, immunogenicity, carcinogenicity, and potential off-target editing. Alternatively, various non-viral vectors such as liposomes, gold nanoparticles, DNA nanostructures, and coordination polymers (CPs) have been wisely designed to deliver gene editing tools for their features such as versatility, tunability, biocompatibility, and high delivery efficiency. Herein, we have sought to design a bio-safe crystalline coordination polymer that could be synthesized, and stored indefinitely under aqueous conditions to ideally function in biological applications. Among all biologically derived organic linkers, carbohydrates are very reasonable choice as biocompatible linker for its polyfunctionality providing good coordination and hydrogen bonding sites. Besides, carbohydrates are abundant, cheap, ecofriendly, and recyclable materials which allow excessive experimental work, and costless translation into clinical trials. In this work, we aimed to design a low dimensional, yet highly biocompatible CPs as carrier for therapeutic biomolecules. The chosen biocompatible components are aldaric acid, namely mucic acid, as the naturally derived organic linker and an endogenous metal, magnesium. we are taking advantage of the structural properties, biocompatibility, and natural targeting ability of carbohydrates. This approach is significantly promising for acquiring a unique biomolecule delivery structure having perfect biocompatibility, crystallinity, and green synthesis. We believe this strategy opens a new bath in the design of biocompatible coordination polymers, answering an important question of what the actual important properties are required for a crystalline coordination polymer to be applied in biological applications.