Abstract
With the generation of induced pluripotent stem cells (iPSCs) derived from adult cells, it is now possible to understand the mechanisms of a genetic disease and potentially identify targets for a disease-specific treatment. It is a long journey from “the bench to the bedside”; however with the available technology, it is possible to identify more suitable treatments than ever before. In this chapter, we will focus on discussing how iPSCs can model a heterogeneous disease known as spinocerebellar ataxia (SCA) and the importance of these studies to understand the disease mechanisms and potentially how a treatment could be achieved.
| Original language | English |
|---|---|
| Title of host publication | Stem Cells in Modeling Human Genetic Diseases |
| Editors | Mayana Zatz, Keith Okamoto |
| Place of Publication | = |
| Publisher | Springer |
| Pages | 45-62 |
| Number of pages | 18 |
| ISBN (Electronic) | 9783319183145 |
| ISBN (Print) | 9783319183138 |
| Publication status | Published - 2015 |
Keywords
- Induced Pluripotent Stem Cells
- SPINOCEREBELLAR ATAXIA
- Modelling
- human disease