Synthesis and characterization of a novel bioresorbable polymer for gene therapy applications

P.C. CARACCIOLO; F. PARRA; G.A. ABRAHAM; B. VÁZQUEZ; J. SAN ROMÁN

Mar del Plata

Taller; 2 Taller de Órganos Artificiales, Biomateriales e Ingeniería de Tejidos; 2011

Universidad Nacional de Mar del Plata

Gene therapy involves the insertion of a functional copy of a defective or absent gene in an individual´s cells and tissues, with the purpose of treating a disease, replacing lost cellular functions or introducing a new function. This therapy has great potential in treating diseases such as cancer and AIDS. To this end, the gene requires a vector to be internalized into the cells and carry out its biological function. The success of this therapy depends largely on the efficiency of the vector. In recent years there has been substantial progress in the development and application of non-viral vectors. However, there are still many problems to be solved before this therapy becomes a standard clinical practice. In this work, we have explored different synthetic routes to obtain bioresorbable polymers suitable for DNA complexation and gene delivery.  A polyurethane based on LDI (L-lysine methyl ester diisocyanate), L-tyrosine and TEG (tetraethylene glycol), as well as a polyester based on TEG and L-glutamic acid, were synthesized. Both polymers have protonable amino groups in their backbone for the subsequent complexation. Polymers were purified by dialysis, and characterized by DSC, FTIR-ATR, GPC, NMR and acid-base titration.