Article

<i>In vivo</i> base editing rescues photoreceptors in a mouse model of retinitis pigmentosa

2022-06-22

Abstract excerpt

Retinitis pigmentosa (RP) is a group of retinal diseases that cause the progressive death of retinal photoreceptor cells and eventually blindness. Mutations in the β-domain of the phosphodiesterase 6 ( Pde6b ) gene are among the most identified causes of autosomal recessive RP. Here, we report a base editing approach in which adeno-associated virus (AAV)-mediated adenine base editor (ABE) delivery to postmitotic...

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Literature Corpus work: 3cfaee51-207e-5017-9bbf-430e6f1f408d
DOI: 10.1101/2022.06.20.496770

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