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Prime Editing Strategy to Install the Mfrp Retinal Degeneration 6 Mutation
Bruna Lopes da Costa1,2, Jorge Pincay2,3, Scott E Brodie3
1Department of Biomedical Engineering, Columbia University, New York, NY, USA.
Abstract:
Mutations in the MFRP (membrane-type frizzled-related protein) gene are associated with a spectrum of ocular diseases. Here, we report on a patient with MFRP-linked autosomal recessive retinitis pigmentosa (arRP) with nanophthalmos who exhibited yellow deposits circumferentially along with far temporal intraretinal pigment migration. In future studies, we plan to explore the amelioration of MFRP-associated phenotypes in patient-specific induced pluripotent stem cell (iPSC)-derived retinal pigment epithelium and in vivo using the classical Mfrprd6 mouse model of RP. To effectively screen gene editing correction approaches for the Mfrprd6 mouse model, we require a strategy to install the desired mutation in the Neuro-2a (N2a) mouse neuroblastoma cell line. In this study, we developed a prime editing strategy for the installation of the Mfrprd6 c.445+3_6AAGTdel mutation.
Insights
Researchers developed a prime editing strategy to introduce a specific mutation into mouse neuroblastoma cells. This method aids in studying gene editing for MFRP-associated eye diseases like retinitis pigmentosa.
Area of Science:
- Genetics
- Ophthalmology
- Molecular Biology
Background:
- Mutations in the MFRP gene cause various ocular diseases.
- Autosomal recessive retinitis pigmentosa (arRP) with nanophthalmos is linked to MFRP gene mutations.
- Patient presented with unique retinal pigmentary changes.
Purpose of the Study:
- To develop a gene editing strategy for the Mfrprd6 mouse model.
- To install the specific Mfrprd6 c.445+3_6AAGTdel mutation in Neuro-2a cells.
- To facilitate screening of gene editing correction approaches for MFRP-associated retinal diseases.
Main Methods:
- Developed a prime editing strategy.
- Targeted the Mfrprd6 c.445+3_6AAGTdel mutation.
- Utilized the Neuro-2a (N2a) mouse neuroblastoma cell line.
Main Results:
- Successfully developed a prime editing strategy.
- Demonstrated the installation of the Mfrprd6 c.445+3_6AAGTdel mutation in N2a cells.
- Established a method for screening gene editing approaches.
Conclusions:
- Prime editing provides a viable strategy for introducing specific mutations in cell lines.
- This method is crucial for advancing research into MFRP-associated ocular diseases.
- Facilitates the development and testing of gene therapies for retinitis pigmentosa.
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