In vivo base editing rescues photoreceptors in a mouse model of retinitis pigmentosa

• 主要な著者: Jing Su (著者), Kaiqin She (著者), Li Song (著者), Xiu Jin (著者), Ruiting Li (著者), Qinyu Zhao (著者), Jianlu Xiao (著者), Danian Chen (著者), Hui Cheng (著者), Fang Lu (著者), Yuquan Wei (著者), Yang Yang (著者)
• フォーマット: 図書
• 出版事項: Elsevier, 2023-03-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

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 the most identified causes of autosomal recessive RP. Clinically, there is no effec...