Development of a Structural Nucleic Acid Delivery Vector Technology
The delivery of biomacromolecules such as proteins and nucleic acids to specific cells inside the body remains a critical challenge in contemporary biomedical research. Delivery technologies to accomplish this are ideally safe, effective, versatile, and scalable, but the current commercial technolog...
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| Format: | Thesis |
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Massachusetts Institute of Technology
2024
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| Online Access: | https://hdl.handle.net/1721.1/155323 https://orcid.org/0000-0002-5041-2383 |
| _version_ | 1811090046953979904 |
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| author | Knappe, Grant Alexander |
| author2 | Bathe, Mark |
| author_facet | Bathe, Mark Knappe, Grant Alexander |
| author_sort | Knappe, Grant Alexander |
| collection | MIT |
| description | The delivery of biomacromolecules such as proteins and nucleic acids to specific cells inside the body remains a critical challenge in contemporary biomedical research. Delivery technologies to accomplish this are ideally safe, effective, versatile, and scalable, but the current commercial technologies often fall short of these characteristics. Nucleic acid nanoparticles, which are computationally-designed nanostructured assemblies of nucleic acid, represent a promising foundational technology to utilize in delivery applications. Here, I present several advancements towards developing a delivery platform designed on nucleic acid nanoparticles. After introducing the current challenges in delivery and the current commercial technologies, I detail why nucleic acid nanoparticles offer promise as components of a delivery technology. Then, I describe my thesis work on evaluating the safety of nucleic acid nanoparticles in pre-clinical animal models, on their fabrication with a focus on integrating new chemistries and characterization techniques, and on a proof-of-concept demonstration in using nucleic acid nanoparticles to deliver viral antigens in a vaccine formulation. Finally, I summarize where I think the state of the technology is today, where its potential could reach, and what the path to reach that potential is. |
| first_indexed | 2024-09-23T14:31:22Z |
| format | Thesis |
| id | mit-1721.1/155323 |
| institution | Massachusetts Institute of Technology |
| last_indexed | 2024-09-23T14:31:22Z |
| publishDate | 2024 |
| publisher | Massachusetts Institute of Technology |
| record_format | dspace |
| spelling | mit-1721.1/1553232024-06-28T03:17:09Z Development of a Structural Nucleic Acid Delivery Vector Technology Knappe, Grant Alexander Bathe, Mark Hammond, Paula T. Massachusetts Institute of Technology. Department of Chemical Engineering The delivery of biomacromolecules such as proteins and nucleic acids to specific cells inside the body remains a critical challenge in contemporary biomedical research. Delivery technologies to accomplish this are ideally safe, effective, versatile, and scalable, but the current commercial technologies often fall short of these characteristics. Nucleic acid nanoparticles, which are computationally-designed nanostructured assemblies of nucleic acid, represent a promising foundational technology to utilize in delivery applications. Here, I present several advancements towards developing a delivery platform designed on nucleic acid nanoparticles. After introducing the current challenges in delivery and the current commercial technologies, I detail why nucleic acid nanoparticles offer promise as components of a delivery technology. Then, I describe my thesis work on evaluating the safety of nucleic acid nanoparticles in pre-clinical animal models, on their fabrication with a focus on integrating new chemistries and characterization techniques, and on a proof-of-concept demonstration in using nucleic acid nanoparticles to deliver viral antigens in a vaccine formulation. Finally, I summarize where I think the state of the technology is today, where its potential could reach, and what the path to reach that potential is. Ph.D. 2024-06-27T19:44:55Z 2024-06-27T19:44:55Z 2024-05 2024-05-16T14:02:34.705Z Thesis https://hdl.handle.net/1721.1/155323 https://orcid.org/0000-0002-5041-2383 In Copyright - Educational Use Permitted Copyright retained by author(s) https://rightsstatements.org/page/InC-EDU/1.0/ application/pdf Massachusetts Institute of Technology |
| spellingShingle | Knappe, Grant Alexander Development of a Structural Nucleic Acid Delivery Vector Technology |
| title | Development of a Structural Nucleic Acid Delivery Vector Technology |
| title_full | Development of a Structural Nucleic Acid Delivery Vector Technology |
| title_fullStr | Development of a Structural Nucleic Acid Delivery Vector Technology |
| title_full_unstemmed | Development of a Structural Nucleic Acid Delivery Vector Technology |
| title_short | Development of a Structural Nucleic Acid Delivery Vector Technology |
| title_sort | development of a structural nucleic acid delivery vector technology |
| url | https://hdl.handle.net/1721.1/155323 https://orcid.org/0000-0002-5041-2383 |
| work_keys_str_mv | AT knappegrantalexander developmentofastructuralnucleicaciddeliveryvectortechnology |