CRISPR-mediated optogene expression from a cell-specific endogenous promoter in retinal ON-bipolar cells to restore vision

Retinitis pigmentosa, an inherited form of retinal degeneration, is characterized by a progressive loss of rods and subsequent degeneration of cones, leading to blindness. However, the remaining neural portion of the retina (bipolar and ganglion cells) remains anatomically and functionally intact fo...

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Κύριοι συγγραφείς:	A. Maddalena, S. Kleinlogel
Μορφή:	Artigo
Γλώσσα:	Inglês
Έκδοση:	Frontiers Media S.A. 2023-03-01
Σειρά:	Frontiers in Drug Delivery
Θέματα:	optogenetics CRISPR/Cas9-mediated genome editing vision restoration gene therapy exo-AAV Opto-mGluR6
Διαθέσιμο Online:	https://www.frontiersin.org/articles/10.3389/fddev.2023.934394/full
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