Bayona-Bafaluy Pilar, Sanz-Pons Javier, Esteban Olivia, Bueno-Borghi Luca, Ruiz-Pesini Eduardo
Departamento de Bioquímica, Biología Molecular y Celular, Universidad de Zaragoza, Zaragoza, Spain.
Instituto de Investigación Sanitaria (IIS) de Aragón, Zaragoza, Spain.
Clin Genet. 2025 May;107(5):505-510. doi: 10.1111/cge.14683. Epub 2024 Dec 23.
An in-depth analysis of susceptibility factors modifying the penetrance of rare Leber hereditary optic neuropathy-causing mutations in respiratory complex I genes encoded in mitochondrial deoxyribonucleic acid has not been performed. To bridge this gap, we conducted a review of the literature on rare mutations associated with LHON, selected those with substantial evidence of pathogenicity, and performed an in-depth analysis of the various pedigrees. Examining the influences that modify the penetrance of the classical mutations associated with this disease may offer insights into susceptibility factors in individuals carrying the rare mutations.
尚未对影响线粒体脱氧核糖核酸中编码的呼吸链复合体I基因的罕见致Leber遗传性视神经病变突变外显率的易感性因素进行深入分析。为了填补这一空白,我们对与Leber遗传性视神经病变相关的罕见突变的文献进行了综述,选择了那些有充分致病性证据的突变,并对各种家系进行了深入分析。研究影响该疾病相关经典突变外显率的因素,可能会为携带罕见突变个体的易感性因素提供见解。