Prime Editing Strategy to Install the Mfrp Retinal Degeneration 6 Mutation.

• Tác giả: Scott E Brodie, Bruna Lopes da Costa, Jorge Pincay, Peter M J Quinn, Stephen H Tsang
• Ngôn ngữ: eng
• Ký hiệu phân loại: 297.1248 Sources of Islam
• Thông tin xuất bản: United States : Advances in experimental medicine and biology , 2025
• Bộ sưu tập: NCBI
• ID: 50030

Tóm tắt

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 Mfrp