# In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

> Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter...

## Metadata
- Authors: Benjamin Bakondi, Wenjian Lv, Bin Lü, Melissa K. Jones, Yu-Chun Tsai, Kevin J. Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J. Breunig, Clive N. Svendsen, Shaomei Wang
- Journal: Molecular Therapy
- Published: 2015-12-15
- DOI: https://doi.org/10.1038/mt.2015.220
- Citations: 290
- Source: OpenAlex
- Access: Open Access

## Technology Hub
- Hub: CRISPR Gene Editing
- Discipline: Biology / Genetics
- Hub URL: https://science-database.com/technology/crispr
- Hub llms.txt: https://science-database.com/technology/crispr/llms.txt

## Abstract
Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter mutation (Rho(S334)) in rats that model severe autosomal dominant retinitis pigmentosa. A single subretinal injection of guide RNA/Cas9 plasmid in combination with electroporation generated allele-specific disruption of Rho(S334), which prevented retinal degeneration and improved visual function.

## Links
- DOI: https://doi.org/10.1038/mt.2015.220
- OpenAlex: https://openalex.org/W2199356015
- PDF: http://www.cell.com/article/S152500161630973X/pdf
- JSON API: https://science-database.com/api/v1/technology/crispr

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Paper ID: oa-W2199356015 | Hub: crispr