Branham Kari, Schlegel Dana, Fahim Abigail T, Jayasundera K Thiran
Department of Ophthalmology and Visual Sciences, University of Michigan, Kellogg Eye Center, Ann Arbor, Michigan, USA.
Am J Med Genet C Semin Med Genet. 2020 Sep;184(3):571-577. doi: 10.1002/ajmg.c.31835. Epub 2020 Aug 31.
Inherited retinal degenerations (IRDs) are a genotypically and phenotypically diverse group of conditions. Great strides have been made toward identifying the genetic basis for these conditions over the last 30 years-more than 270 different genes involved in syndromic and nonsyndromic forms of retinal dystrophies have now been identified. The identification of these genes and the improvement of clinical laboratory techniques have led to the identification of the genetic basis of disease in 56-76% of patients with IRDs through next generation sequencing and copy number variant analysis. Genetic testing is an essential part of clinical care for patients affected with IRDs and is required to confirm the diagnosis, understand the inheritance of the condition, and determine eligibility for gene-specific treatments or clinical trials. Despite the success achieved in determining the genetic cause of these conditions, several challenges remain, which must be considered when providing genetic testing and genetic counseling to patients. For this reason, an integrated team of ophthalmic and genetic clinicians who are familiar with these challenges is necessary to provide optimal comprehensive care to these patients.
遗传性视网膜变性(IRD)是一组在基因型和表型上具有多样性的疾病。在过去30年里,在确定这些疾病的遗传基础方面取得了巨大进展——目前已鉴定出270多个与综合征性和非综合征性视网膜营养不良形式相关的不同基因。这些基因的鉴定以及临床实验室技术的改进,通过下一代测序和拷贝数变异分析,在56%-76%的IRD患者中确定了疾病的遗传基础。基因检测是IRD患者临床护理的重要组成部分,对于确诊、了解疾病的遗传方式以及确定是否适合进行基因特异性治疗或临床试验都必不可少。尽管在确定这些疾病的遗传原因方面取得了成功,但仍存在一些挑战,在为患者提供基因检测和遗传咨询时必须加以考虑。因此,需要一个由熟悉这些挑战的眼科和遗传临床医生组成的综合团队,为这些患者提供最佳的综合护理。