Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas.

"deep sequencing data are available from the ncbi sequence read archive under accession number prjna784338 . 0 02% ( data s1 a ) and 0 04 ?; 0 02% ( data s1 b ) and 0 12 ?; notably no apparent editing activities were found at the sites of ngrna ( data s1 c and d ).; all pegrna and ngrna constructs were generated by ligation of annealed oligos into pu6 expression plasmids and cloned into the modified paav-ef1?-gfp-p2a-spry(h840a) 1-713 -npun with kpni and nhei restriction sites for the insertions of u6-pegrna and u6-ngrna cassettes (see supplemental sequences and notes in data s2 ).; data s1 show the sequences of indels for the pegrna or ngrna in the treated retinas.; data s2 show the sequences of constructs used in this study.; supplemental notes in data s2 show the cloning protocol of the constructs.; data s1 shows sequences of indels for the pegrna or ngrna in the treated retinas (a and b) the alleles of the top ten indels from genomic dna of retinas subjected to pe spry system-mediated pde6b t to c (a) and pde6b aga (b) editing with the p5n3 combination and the optimized pbs and rt template.; data s2 show the sequences of constructs used in this study."

"Disclosures: The authors declare no competing interests exist."

"This work was supported by the National Natural Science Foundation of China (no. 31970930, no. 32100936), the Hubei Natural Science Foundation (no. 2020CFA069, no. 2018CFB434), and the Neuroscience Team Development Project of Wuhan University of Science and Technology (no. 1180002)."