dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy

Lisa M. Riedmayr, Klara S. Hinrichsmeyer, Nina Karguth, Sybille Böhm, Victoria Splith, Stylianos Michalakis, Elvir Becirovic

Published: 2022-02-06 DOI: 10.1038/s41596-021-00666-3

gene therapy

transcriptional activation

dCas9-VPR

functionally equivalent genes

inherited retinal diseases

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Many disease-causing genes possess functionally equivalent counterparts, which are often expressed in distinct cell types. An attractive gene therapy approach for inherited disorders caused by mutations in such genes is to transcriptionally activate the a
Introduction
Materials
Procedure
Troubleshooting
Timing
Anticipated results
- Reporting Summary
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References
ACKNOWLEDGMENTS

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dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy

Supplementary information

Supplementary Table 1

Reporting Summary

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