Biology

Title

Functionalization of Linear Poly(ethylenimine) as DNA-Binding Polymer for Gene Therapy Application

Date of Award

6-2017

Degree Type

Thesis

Degree Name

Master of Science in Biochemistry & Molecular Biology

Department

Biology

Chief Instructor

Sergio Granados-Focil

Second Reader

Rob Bellin

Third Reader

Denis A. Larochelle

Abstract

Gene therapy is a technique used to combat diseases by correcting the defective gene responsible for the disease to alleviate its symptoms and restore normal function to the defective gene. Gene therapy uses vectors to deliver a functional copy of the defective gene targeted towards the defective cell to replace the defective gene with the functional one via gene transfer (1). Over the years, gene delivery vectors available for gene therapy have gradually evolved, leading to the development of new and better vectors for gene delivery. These advances contribute largely to the growing successes of gene therapy and its application (2). There are two types of vectors available for gene delivery: viral and non-viral vectors. Although viral vector systems have shown high efficiencies in delivering genes, genome insertion, and gene expression within cells, the lack of control over where the integration of the therapeutic gene occurs poses significant safety risks. The significant safety risks posed by viral vectors has led to the investigation of using non-viral vectors as potential gene delivery vehicles. Their delivery mechanisms can be better controlled, compared to their viral vectors counterparts. A particularly attractive target for future development is cationic polymers because their architecture can be altered to enhance the polymer's function, such as reduced toxicity and polymer aggregation within cells. Among these cationic polymers, polyethyleneimine (PEI) has shown promising DNA complexation and transfection efficiency but has also exhibited high toxicity in cells.

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