INTEMA   05428
INSTITUTO DE INVESTIGACIONES EN CIENCIA Y TECNOLOGIA DE MATERIALES
Unidad Ejecutora - UE
congresos y reuniones científicas
Título:
Synthesis and characterization of a novel bioresorbable polymer for gene therapy applications
Autor/es:
P.C. CARACCIOLO; F. PARRA; G.A. ABRAHAM; B. VÁZQUEZ; J. SAN ROMÁN
Lugar:
Mar del Plata
Reunión:
Taller; 2° Taller de Órganos Artificiales, Biomateriales e Ingeniería de Tejidos (OBI 2011); 2011
Institución organizadora:
INTEMA
Resumen:
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. 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 obtained a bioresorbable polymer suitable for DNA complexation and gene delivery. A polyester based on tetraethylene glycol and L-glutamic acid was synthesized, employing piro L-glutamic acid and amino group protection with ditert butyl dicarbonate. The polymers were purified by dialysis, and characterized by TGA, DSC, FTIR-ATR, GPC, NMR and acid-base titration. The material resulted amorphous and rubbery at physiological temperature. The protonated amino groups could perform DNA complexation, while the material could be able to facilitate endosomal escape of polymer-DNA polyplexes, due to its buffering capacity. Thus, this polymer could be suitable for DNA complexation and gene delivery.