The use of induced pluripotent stem cells to reveal pathogenic gene mutations and explore treatments for retinitis pigmentosa
Summary
The use of RP patient-derived iPSCs combined with genome editing provided a versatile cellular system with which to define the roles of genetic mutations in isogenic iPSCs with or without mutation and also provided a system that can be used to explore candidate therapeutic approaches.
| Authors | Yoshida T, Ozawa Y, Suzuki K, Yuki K, Ohyama M, Akamatsu W, Matsuzaki Y, Shimmura S, Mitani K, Tsubota K, Okano H |
|---|---|
| Journal | Molecular brain |
| Publication Date | 2014 Jun 16;7:45 |
| PubMed | 24935155 |
| PubMed Central | PMC4058693 |
| DOI | 10.1186/1756-6606-7-45 |