Regulable and cell-type-specific rescue of Niemann-pick disease type C, a neurodegenerative lysosomal storage disorder
Abstract/Contents
- Abstract
- Niemann-Pick disease type C (NPC) is a rare metabolic lysosomal storage disorder (LSD) marked by accumulation of large quantities of cholesterol, lipids and other metabolites in cells. Though the disorder has been extensively explored using various genetic animal models, an understanding of the molecular and cellular pathology of the disease remains limited. A cell-type-specific and regulable rescue mouse model of NPC disease was engineered in order to identify the therapeutically relevant cell type and ascertain the effect of inflammation on disease progression. With the current information presented in this dissertation, a probable road map of NPC disease pathology has been drawn. The road map for NPC may also be applicable to, or act as a template for, other lysosomal storage diseases and neurodegenerative disorders with similar pathologies.
Description
| Type of resource | text |
|---|---|
| Form | electronic; electronic resource; remote |
| Extent | 1 online resource. |
| Publication date | 2011 |
| Issuance | monographic |
| Language | English |
Creators/Contributors
| Associated with | Lopez, Manuel Eduardo |
|---|---|
| Associated with | Stanford University, Department of Developmental Biology. |
| Primary advisor | Scott, Matthew P |
| Thesis advisor | Scott, Matthew P |
| Thesis advisor | Barres, Ben |
| Thesis advisor | Luo, Liqun, 1966- |
| Advisor | Barres, Ben |
| Advisor | Luo, Liqun, 1966- |
Subjects
| Genre | Theses |
|---|
Bibliographic information
| Statement of responsibility | Manuel Eduardo Lopez, Jr. |
|---|---|
| Note | Submitted to the Department of Developmental Biology. |
| Thesis | Ph.D. Stanford University 2011 |
| Location | electronic resource |
Access conditions
- Copyright
- © 2011 by Manuel Eduardo Lopez
- License
- This work is licensed under a Creative Commons Attribution Non Commercial 3.0 Unported license (CC BY-NC).
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