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Journal article

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

Abstract:

Rhodopsin (RHO) gene mutations are a common cause of autosomal dominant retinitis pigmentosa (ADRP). The need to suppress toxic protein expression together with mutational heterogeneity pose challenges for treatment development. Mirtrons are atypical RNA interference effectors that are spliced from transcripts as short introns. Here, we develop a novel mirtron-based knockdown/replacement gene therapy for the mutation-independent treatment of RHO-related ADRP, and demonstrate efficacy in a rel...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1038/s41467-021-25204-3

Authors


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Role:
Author
ORCID:
0000-0002-9770-6303
More by this author
Institution:
University of Oxford
Division:
MSD
Sub department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0002-2941-4464
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Research group:
Clinical Ophthalmology Research Group
Role:
Author
ORCID:
0000-0002-0662-1073
More by this author
Institution:
University of Oxford
Division:
MSD
Sub department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0002-6612-6162
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Research group:
Clinical Ophthalmology Research Group
Oxford college:
Merton College
Role:
Author
ORCID:
0000-0002-3096-4682
Royal College of Surgeons Edinburgh More from this funder
Royal College of Surgeons Edinburgh More from this funder
Medical Research Council More from this funder
Publisher:
Springer Nature Publisher's website
Journal:
Nature Communications Journal website
Volume:
12
Issue:
1
Article number:
4934
Place of publication:
England
Publication date:
2021-08-16
Acceptance date:
2021-07-26
DOI:
EISSN:
2041-1723
Pmid:
34400638

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