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

Resumen

The integration of novel compounds into formulations may open up new avenues andopportunities for improving the treatment of various diseases using a gene therapytechnique. To this purpose, my thesis aimed to study the impact of incorporation of biomaterialssuch sphingolipids or nanodiamonds as a helper component into a cationic niosome formulationfor 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 traffickingpathways, and transfection efficiency.