Advancing RNA therapeutics : regulation, immunogenicity, and delivery
Abstract/Contents
- Abstract
- The Central Dogma of molecular biology formalized the significant role of RNA in regulating the flow of information from DNA to proteins. However, beyond its canonical function in translation, RNA carries a wide array of functions, serving as a molecular machinery that regulates gene expression during development and cellular differentiation. Furthermore, the remarkable success of RNA-based therapies in combating the COVID-19 pandemic has sparked greater interest in the translational potential of this remarkable molecule. The renewed enthusiasm in RNA medicine comes from its simple design, safe and relatively fast manufacturing, and, most importantly, its diverse molecular formats, capable of reaching formerly deemed 'undruggable' targets at the DNA, RNA, or protein levels. Furthermore, the large variety of RNA molecules, including the most recently described class, known as circular RNA, uncovers many new biological functions and therapeutic applications. Nevertheless, the development of RNA therapeutics still remains hampered by significant challenges. These encompass the rapid degradation of exogenous RNA by ubiquitous RNases, the difficulty of transporting negatively charged RNA through hydrophobic cytoplasmic membranes, and the potent immunogenicity of exogenous RNA, leading to cellular toxicity and hindering its translation into therapeutic proteins. My dissertation addresses these hurdles, exploring mechanisms of RNA delivery for vaccine development and cell reprogramming. Chapters delve into circular RNA uptake, immunogenicity, and biodistribution, proposing novel nanoparticle transporters for targeted mRNA and circular RNA delivery. The research aims to transform vaccine development and cell therapy by enhancing circRNA delivery.
Description
| Type of resource | text |
|---|---|
| Form | electronic resource; remote; computer; online resource |
| Extent | 1 online resource. |
| Place | California |
| Place | [Stanford, California] |
| Publisher | [Stanford University] |
| Copyright date | 2023; ©2023 |
| Publication date | 2023; 2023 |
| Issuance | monographic |
| Language | English |
Creators/Contributors
| Author | Amaya Hernandez, Laura Cristina |
|---|---|
| Degree supervisor | Chang, Howard Y. (Howard Yuan-Hao), 1972- |
| Thesis advisor | Chang, Howard Y. (Howard Yuan-Hao), 1972- |
| Thesis advisor | Majeti, Ravindra, 1972- |
| Thesis advisor | Pulendran, B. (Bali) |
| Degree committee member | Majeti, Ravindra, 1972- |
| Degree committee member | Pulendran, B. (Bali) |
| Associated with | Stanford University, School of Medicine |
| Associated with | Stanford University, Department of Stem Cell Biology and Regenerative Medicine |
Subjects
| Genre | Theses |
|---|---|
| Genre | Text |
Bibliographic information
| Statement of responsibility | Laura Amaya. |
|---|---|
| Note | Submitted to the Department of Stem Cell Biology and Regenerative Medicine. |
| Thesis | Thesis Ph.D. Stanford University 2023. |
| Location | https://purl.stanford.edu/kz329dd2556 |
Access conditions
- Copyright
- © 2023 by Laura Cristina Amaya Hernandez
- License
- This work is licensed under a Creative Commons Attribution Non Commercial 3.0 Unported license (CC BY-NC).
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