Abstract：
Inherited retinal dystrophies and retinal degenerations related to more common diseases (i.e., age-related macular dystrophy) are a major issue and one of the main causes of low vision in pediatric and elderly age groups. Advancement and understanding in molecular biology and the possibilities raised by gene-editing techniques opened a new era for clinicians and patients due to feasible possibilities of treating disabling diseases and the reduction in their complications burden. The scope of this review is to focus on the state-of-the-art in somatic cell therapy medicinal products as the basis of new insights and possibilities to use this approach to treat rare eye diseases.
摘要：
与更常见的疾病相关的遗传性视网膜营养不良和视网膜变性(即，年龄相关性黄斑营养不良）是一个主要问题，也是儿童和老年人视力低下的主要原因之一。分子生物学的进步和理解以及基因编辑技术带来的可能性为临床医生和患者开辟了一个新时代，因为治疗致残疾病的可行可能性和减少其并发症负担。本综述的范围是专注于最先进的体细胞治疗药物产品，作为使用这种方法治疗罕见眼病的新见解和可能性的基础。
