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一种罕见病症的基因分析:一个白种人家庭中白化病与多发性原发性黑色素瘤的共现情况。

Genetic profiling of a rare condition: co-occurrence of albinism and multiple primary melanoma in a Caucasian family.

作者信息

De Summa Simona, Guida Michele, Tommasi Stefania, Strippoli Sabino, Pellegrini Cristina, Fargnoli Maria Concetta, Pilato Brunella, Natalicchio Iole, Guida Gabriella, Pinto Rosamaria

机构信息

IRCCS Istituto Tumori "Giovanni Paolo II", Molecular Genetics Laboratory, Bari, Italy.

IRCCS Istituto Tumori "Giovanni Paolo II", Oncology Unit, Bari, Italy.

出版信息

Oncotarget. 2017 May 2;8(18):29751-29759. doi: 10.18632/oncotarget.12777.

Abstract

Multiple primary melanoma (MPM) is a rare condition, whose genetic basis has not yet been clarified. Only 8-12% of MPM are due to germline mutations of CDKN2A. However, other genes (POT1, BRCA1/2, MC1R, MGMT) have been demonstrated to be involved in predisposition to this pathology.To our knowledge, this is the first family study based on two siblings with the rare coexistence of MPM and oculocutaneous albinism (OCA), an autosomal recessive disease characterized by the absence or decrease in pigmentation in the skin, hair, and eyes.In this study, we evaluated genes involved in melanoma predisposition (CDKN2A, CDK4, MC1R, MITF, POT1, RB1, MGMT, BRCA1, BRCA2), pathogenesis (BRAF, NRAS, PIK3CA, KIT, PTEN), skin/hair pigmentation (MC1R, MITF) and in immune pathways (CTLA4) to individuate alterations able to explain the rare onset of MPM and OCA in indexes and the transmission in their pedigree.From the analysis of the pedigree, we were able to identify a "protective" haplotype with respect to MPM, including MGMT p.I174V alteration. The second generation offspring is under strict follow up as some of them have a higher risk of developing MPM according to our model.

摘要

多发性原发性黑色素瘤(MPM)是一种罕见疾病,其遗传基础尚未明确。仅8%-12%的MPM是由CDKN2A的种系突变引起的。然而,已证实其他基因(POT1、BRCA1/2、MC1R、MGMT)与该疾病的易感性有关。据我们所知,这是第一项基于两名罕见地同时患有MPM和眼皮肤白化病(OCA)的兄弟姐妹的家族研究,OCA是一种常染色体隐性疾病,其特征是皮肤、头发和眼睛色素沉着缺失或减少。在本研究中,我们评估了与黑色素瘤易感性(CDKN2A、CDK4、MC1R、MITF、POT1、RB1、MGMT、BRCA1、BRCA2)、发病机制(BRAF、NRAS、PIK3CA、KIT、PTEN)、皮肤/头发色素沉着(MC1R、MITF)以及免疫途径(CTLA4)相关的基因,以确定能够解释先证者中MPM和OCA罕见发病及其家系遗传的改变。通过对家系的分析,我们能够识别出一种相对于MPM的“保护性”单倍型,包括MGMT p.I174V改变。根据我们的模型,第二代后代受到严格随访,因为他们中的一些人患MPM的风险更高。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1b3e/5444700/fe5f41bfa176/oncotarget-08-29751-g001.jpg

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