| Title: |
Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids |
| Authors: |
Lane A.; Jovanovic K.; Shortall C.; Ottaviani D.; Panes A. B.; Schwarz N.; Guarascio R.; Hayes M. J.; Palfi A.; Chadderton N.; Farrar G. J.; Hardcastle A. J.; Cheetham M. E. |
| Contributors: |
Lane, A.; Jovanovic, K.; Shortall, C.; Ottaviani, D.; Panes, A. B.; Schwarz, N.; Guarascio, R.; Hayes, M. J.; Palfi, A.; Chadderton, N.; Farrar, G. J.; Hardcastle, A. J.; Cheetham, M. E. |
| Publisher Information: |
Cell Press |
| Publication Year: |
2020 |
| Collection: |
Padua Research Archive (IRIS - Università degli Studi di Padova) |
| Subject Terms: |
AAV; cell death; CRISPR gene editing; disease modeling; gene therapy; genetic; inherited disease; retinal degeneration; retinal organoid; stem cell |
| Description: |
Cheetham and colleagues show that 3D retinal organoids lacking the RP2 protein develop rod photoreceptor degeneration that can be prevented with AAV gene augmentation for RP2. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/32531192; info:eu-repo/semantics/altIdentifier/wos/WOS:000550033500007; volume:15; issue:1; firstpage:67; lastpage:79; numberofpages:13; journal:STEM CELL REPORTS; https://hdl.handle.net/11577/3367760 |
| DOI: |
10.1016/j.stemcr.2020.05.007 |
| Availability: |
https://hdl.handle.net/11577/3367760; https://doi.org/10.1016/j.stemcr.2020.05.007 |
| Rights: |
info:eu-repo/semantics/openAccess ; license:Creative commons ; license uri:http://creativecommons.org/licenses/by/4.0/ |
| Accession Number: |
edsbas.C405B70B |
| Database: |
BASE |