Targeting the photoreceptor cilium for the treatment of retinal diseases

被引：18

作者：

Ran, Jie ^{[1
]}

Zhou, Jun ^{[1
,2
]}

机构：

[1] Shandong Normal Univ, Collaborat Innovat Ctr Cell Biol Univ Shandong, Shandong Prov Key Lab Anim Resistance Biol, Inst Biomed Sci,Coll Life Sci, Jinan 250014, Peoples R China

[2] Nankai Univ, Coll Life Sci, State Key Lab Med Chem Biol, Tianjin 300071, Peoples R China

来源：

ACTA PHARMACOLOGICA SINICA | 2020年 / 41卷 / 11期

基金：

中国国家自然科学基金;

关键词：

retina; photoreceptor cilium; retinal disease; ciliopathy; gene therapy; genome editing technology; stem cell-based therapy; HDAC6; inhibitor; LEBER CONGENITAL AMAUROSIS; SENIOR-LOKEN SYNDROME; CONE-ROD DYSTROPHY; JOUBERT SYNDROME; GENE-THERAPY; MUTATIONS; PROTEIN; TRANSPORT; TUBULIN; MICE;

D O I：

10.1038/s41401-020-0486-3

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

Photoreceptors, as polarised sensory neurons, are essential for light sensation and phototransduction, which are highly dependent on the photoreceptor cilium. Structural defects and/or dysfunction of the photoreceptor cilium caused by mutations in photoreceptor-specific genes or common ciliary genes can lead to retinal diseases, including syndromic and nonsyndromic diseases. In this review, we describe the structure and function of the photoreceptor cilium. We also discuss recent findings that underscore the dysregulation of the photoreceptor cilium in various retinal diseases and the therapeutic potential of targeting ciliary genes in these diseases.

引用

页码：1410 / 1415

页数：6

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