体细胞疗法药物治疗遗传性视网膜营养不良：综述
Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review.
作者信息
Bacci Giacomo Maria, Becherucci Valentina, Marziali Elisa, Sodi Andrea, Bambi Franco, Caputo Roberto
机构信息
Pediatric Ophthalmology Unit, Children's Hospital A. Meyer-University of Florence, 50139 Florence, Italy.
Cell Factory Meyer, Children's Hospital A. Meyer-University of Florence, 50139 Florence, Italy.
出版信息
Life (Basel). 2022 May 9;12(5):708. doi: 10.3390/life12050708.
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.
遗传性视网膜营养不良以及与更常见疾病（如年龄相关性黄斑营养不良）相关的视网膜变性是一个主要问题，也是儿童和老年人群低视力的主要原因之一。分子生物学的进展与认识以及基因编辑技术带来的可能性，为临床医生和患者开启了一个新时代，因为治疗致残性疾病以及减轻其并发症负担成为可行的可能性。本综述的范围是聚焦于体细胞治疗药物产品的最新进展，以此作为利用这种方法治疗罕见眼病的新见解和可能性的基础。
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