Title:Current Non-viral Gene Therapy Strategies for the Treatment of Glioblastoma
Volume: 28
Issue: 37
Author(s): Antonela Sofía Asad, Alejandro Javier Nicola Candia, Nazareno González, Camila Florencia Zuccato, Adriana Seilicovich and Marianela Candolfi*
Affiliation:
- Instituto de Investigaciones Biomedicas (INBIOMED, UBA-CONICET), Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires,Argentina
Keywords:
Glioblastoma, non-viral gene therapy, cationic lipids, dendrimers, dendrigrafts, polymeric micelles, poly
(β-amino esters), sleeping beauty transposons.
Abstract:
Background: Glioblastoma constitutes the most frequent and aggressive primary
malignant brain tumor in adults. Despite the advances in its treatment, its prognosis
remains very poor. Gene therapy has been proposed as a complementary treatment since
it may overcome the problem of the blood-brain barrier for systemic therapies, allowing
to target tumor cells and their tumor microenvironment locally, without affecting the normal
brain parenchyma. In comparison with viral vectors, non-viral vectors became an attractive
tool due to their reduced potential of biosafety risks, lower cost, higher availability,
and easy storage.
Objective: In this article, we aimed to outline the current preclinical and clinical developments
of non-viral delivery systems for therapeutic transgene delivery in malignant gliomas.
Conclusion: Non-viral vectors are efficient tools for gene delivery since they exhibit reduced
non-specific cytotoxicity and can go through several modifications in order to
achieve high tumor tropism and the ability to cross the blood-brain barrier to access the
tumor mass. However, further evaluations in preclinical models and clinical trials are required
in order to translate it into the neuro-oncology clinic.
