Home
Resource
Multifunctional nanotechnology approaches for advancing gene therapy
PDF
PDF format is widely accepted and good for printing.
Plug-in required
PDF-1(18.95 MB)
Citation & Export
View Usage Statistics
Staff View

Citation & Export
Hide

Simple citation

Pongkulapa, Thanapat. Multifunctional nanotechnology approaches for advancing gene therapy. Retrieved from https://doi.org/doi:10.7282/t3-nssf-r852

Export

Click here for information about Citation Management Tools at Rutgers.

Statistics
Hide

Description

TitleMultifunctional nanotechnology approaches for advancing gene therapy
NamePongkulapa, Thanapat (author); Lee, Ki-Bum (chair); Warmuth, Ralf (member); Khare, Sagar (member); Gervasoni, Jr., James E. (member); Rutgers University; School of Graduate Studies
Date Created2022
Other Date2022-01 (degree)
SubjectChemistry, Nanotechnology, Cancer, Gene delivery, Gene therapy, Genetic disorders, Magnetic core-shell nanoparticle, Nucleic acid nanotechnology
Extent130 pages
DescriptionThe rising demand for complex biological therapies (i.e., personalized medicine) has created an urgent need for robust gene therapy techniques that permit high therapeutic outcomes with low adverse side effects. Nanotechnology has shown to be an excellent toolkit for advocating the development of gene therapies, especially as a gene carrier allowing the delivery of therapeutic genes into the target locations with minimizing non-specific delivery. Although nanotechnology and nanomaterials have been used widely as drug carriers, their full potential is yet evidenced in clinically relevant models. To this end, this dissertation will focus on how advanced nanotechnology approaches can be adopted to address multiple challenges in gene therapy for the potential treatment of diseases with genetic components (i.e., neurological genetic disorders, cancers, metabolic diseases). Specifically, in two chapters of this dissertation, we will focus on magnetic core-shell nanoparticles for drug and gene delivery. In the first chapter, these nanoparticles are used to deliver microRNA to overcome chemoresistance in cancer. The surface properties of this material can be easily tuned for therapeutic loading and modified with a cancer-specific ligand to achieve target-specific delivery to cancer tissue in an animal model. While the magnetic properties are utilized in molecular imaging (i.e., Magnetic Resonance Imaging [MRI]). In the second chapter, we demonstrated the use of the magnetic properties to enhance transfection and perform magnetic-assisted cell sorting within a single platform to improve gene editing efficiency in Rett’s Syndrome patient-derived stem cells. Altogether, the results demonstrate the feasibility of using nanomaterials to improve gene therapy outcomes in in vivo and ex vivo models. The last chapter of this dissertation focuses on another challenge in the clinical translation of nanotechnology-based gene carriers, namely biodegradability. This chapter demonstrates a novel approach to designing a gene carrier with high gene loading capacity, high stability, and biodegradability using nucleic acid-based nanotechnology. Overall, this dissertation covers the nanotechnology approaches that offer the unique benefits toward advancing gene therapy outcomes for clinical applications.
NotePh.D.
NoteIncludes bibliographical references
Genretheses
Persistent URLhttps://doi.org/doi:10.7282/t3-nssf-r852
LanguageEnglish
CollectionSchool of Graduate Studies Electronic Theses and Dissertations
Organization NameRutgers, The State University of New Jersey
RightsThe author owns the copyright to this work.
Version 8.5.5
Rutgers University Libraries - Copyright ©2024