J Clin Invest. 2019 Feb 1;129(2):513-515. doi: 10.1172/JCI126205. Epub 2019 Jan 22.
Achromatopsia is an inherited retinal degeneration characterized by the loss of cone photoreceptor function. In this issue of the JCI, Moshiri et al. characterize a naturally occurring model of the disease in the rhesus macaque caused by homozygous mutations in the phototransduction enzyme PDE6C. Using retinal imaging, and electrophysiologic and biochemical methods, the authors report a clinical phenotype nearly identical to the human condition. These findings represent the first genetic nonhuman primate model of an inherited retinal disease, and provide an ideal testing ground for the development of novel gene replacement, gene editing, and cell replacement therapies for cone dystrophies.
色盲是一种遗传性视网膜变性疾病,其特征是锥光感受器功能丧失。在本期 JCI 中,Moshiri 等人描述了恒河猴中一种由光转导酶 PDE6C 同源突变引起的疾病的自然发生模型。作者使用视网膜成像以及电生理和生化方法,报告了一种与人类疾病几乎完全相同的临床表型。这些发现代表了遗传性视网膜疾病的首个遗传的非人类灵长类动物模型,为开发用于治疗锥细胞营养不良的新型基因替代、基因编辑和细胞替代疗法提供了理想的试验场。