Developing tools for pluripotent stem cell-based therapies using AAV6/Cas9-mediated genome editing
Abstract/Contents
- Abstract
- The application of AAV6/Cas9-mediated genome editing to human pluripotent stem cells (hPSCs) offers the opportunity for efficient in vitro disease modeling, correction of disease-causing mutations, and a platform for engineering novel properties for clinical applications. Here we describe how to edit the genome of hPSCs, ranging from modifying single base pairs to the integration of large transgene cassettes. We then show how with this protocol we can build safety systems to minimize the risk of hPSC-derived teratoma formation. We show how orthogonal safety switches can be used prevent and intervene in the case of an adverse event following the transplantation of hPSC-derived cells. We also demonstrated the utility of this technology by creating isogenic series of the Huntington CAG repeat to model the pathogenic phenotype in vitro. Finally, we show how we can engineer hPSCs to secrete neurotrophic factors such as BDNF and GDNF that have been shown to ameliorate the symptoms of the disease
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 | 2020; ©2020 |
| Publication date | 2020; 2020 |
| Issuance | monographic |
| Language | English |
Creators/Contributors
| Author | Martin, Renata |
|---|---|
| Degree supervisor | Porteus, Matthew H |
| Thesis advisor | Porteus, Matthew H |
| Thesis advisor | Cyert, Martha S, 1958- |
| Thesis advisor | Loh, Kyle |
| Thesis advisor | Palmer, Theo |
| Degree committee member | Cyert, Martha S, 1958- |
| Degree committee member | Loh, Kyle |
| Degree committee member | Palmer, Theo |
| Associated with | Stanford University, Department of Stem Cell Biology and Regenerative Medicine. |
Subjects
| Genre | Theses |
|---|---|
| Genre | Text |
Bibliographic information
| Statement of responsibility | Renata Moura Martin |
|---|---|
| Note | Submitted to the Department of Stem Cell Biology and Regenerative Medicine |
| Thesis | Thesis Ph.D. Stanford University 2020 |
| Location | electronic resource |
Access conditions
- Copyright
- © 2020 by Renata Martin
- License
- This work is licensed under a Creative Commons Attribution Non Commercial 3.0 Unported license (CC BY-NC).
Also listed in
Loading usage metrics...