Evaluation of the biophysical performance upon the integration of helper components into cationic niosomes for the treatment of both retinal and brain diseases by genetherapy approach

Abstract

The integration of novel compounds into formulations may open up new avenues and opportunities for improving the treatment of various diseases using a genetherapytechnique. To this purpose, my thesis aimed to study the impact of incorporation of biomaterials such sphingolipids or nanodiamonds as a helper component into a cationic niosome formulation for gene delivery, the formulations and related complexes were studied in terms of particle size,surface charge, dispersity, and morphology. Furthermore, biological research were conducted inboth in vitro and in vivo experimental trials to evaluate cellular uptake, intracellular trafficking pathways, and transfection efficiency.