{"title":"艾米和朋友们:改善受DNA修复障碍影响的个体的生活。","authors":"Jayne Hughes, Donata Orioli, Lavinia Arseni","doi":"10.1002/1873-3468.70049","DOIUrl":null,"url":null,"abstract":"<p>DNA repair disorders are rare genetic conditions characterized by defects in the mechanisms responsible for repairing damaged DNA. DNA damage occurs frequently due to environmental factors, and in healthy cells, repair systems fix this damage to maintain genomic integrity. In individuals with DNA repair disorders, these mechanisms are impaired, leading to accumulated damage, cellular dysfunction, premature aging, and cell death. Symptoms vary depending on the specific repair pathway defect, with examples including Cockayne syndrome (CS), trichothiodystrophy (TTD), and xeroderma pigmentosum. <i>Amy and Friends</i> was founded by Jayne Hughes in the UK in 2007 to support children/young adults and families suffering from CS, TTD, and linked DNA repair disorders. In this second of a new series on patient advocacy, <i>FEBS Letters</i> interviews Founder and CEO Jayne Hughes and molecular geneticist and specialist team member Prof. Donata Orioli on the aims, achievements, and activities of <i>Amy and Friends</i>.</p>","PeriodicalId":12142,"journal":{"name":"FEBS Letters","volume":"599 10","pages":"1339-1345"},"PeriodicalIF":3.0000,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/1873-3468.70049","citationCount":"0","resultStr":"{\"title\":\"Amy and Friends: improving the lives of individuals affected by DNA repair disorders\",\"authors\":\"Jayne Hughes, Donata Orioli, Lavinia Arseni\",\"doi\":\"10.1002/1873-3468.70049\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>DNA repair disorders are rare genetic conditions characterized by defects in the mechanisms responsible for repairing damaged DNA. DNA damage occurs frequently due to environmental factors, and in healthy cells, repair systems fix this damage to maintain genomic integrity. In individuals with DNA repair disorders, these mechanisms are impaired, leading to accumulated damage, cellular dysfunction, premature aging, and cell death. Symptoms vary depending on the specific repair pathway defect, with examples including Cockayne syndrome (CS), trichothiodystrophy (TTD), and xeroderma pigmentosum. <i>Amy and Friends</i> was founded by Jayne Hughes in the UK in 2007 to support children/young adults and families suffering from CS, TTD, and linked DNA repair disorders. In this second of a new series on patient advocacy, <i>FEBS Letters</i> interviews Founder and CEO Jayne Hughes and molecular geneticist and specialist team member Prof. Donata Orioli on the aims, achievements, and activities of <i>Amy and Friends</i>.</p>\",\"PeriodicalId\":12142,\"journal\":{\"name\":\"FEBS Letters\",\"volume\":\"599 10\",\"pages\":\"1339-1345\"},\"PeriodicalIF\":3.0000,\"publicationDate\":\"2025-05-05\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/epdf/10.1002/1873-3468.70049\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"FEBS Letters\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/1873-3468.70049\",\"RegionNum\":4,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"Biochemistry, Genetics and Molecular Biology\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"FEBS Letters","FirstCategoryId":"99","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/1873-3468.70049","RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"Biochemistry, Genetics and Molecular Biology","Score":null,"Total":0}
引用次数: 0
摘要
DNA修复障碍是一种罕见的遗传疾病,其特征是负责修复受损DNA的机制存在缺陷。由于环境因素,DNA损伤经常发生,在健康细胞中,修复系统修复这种损伤以保持基因组的完整性。在DNA修复紊乱的个体中,这些机制受损,导致累积损伤、细胞功能障碍、过早衰老和细胞死亡。症状取决于特定的修复途径缺陷,包括Cockayne综合征(CS)、毛硫营养不良症(TTD)和色素性干皮病。杰恩·休斯于2007年在英国创立了Amy and Friends,旨在帮助患有CS、TTD和相关DNA修复障碍的儿童/年轻人和家庭。在患者权益新系列的第二部分中,FEBS Letters采访了创始人兼首席执行官Jayne Hughes和分子遗传学家兼专家团队成员Donata Orioli教授关于Amy and Friends的目标、成就和活动。
Amy and Friends: improving the lives of individuals affected by DNA repair disorders
DNA repair disorders are rare genetic conditions characterized by defects in the mechanisms responsible for repairing damaged DNA. DNA damage occurs frequently due to environmental factors, and in healthy cells, repair systems fix this damage to maintain genomic integrity. In individuals with DNA repair disorders, these mechanisms are impaired, leading to accumulated damage, cellular dysfunction, premature aging, and cell death. Symptoms vary depending on the specific repair pathway defect, with examples including Cockayne syndrome (CS), trichothiodystrophy (TTD), and xeroderma pigmentosum. Amy and Friends was founded by Jayne Hughes in the UK in 2007 to support children/young adults and families suffering from CS, TTD, and linked DNA repair disorders. In this second of a new series on patient advocacy, FEBS Letters interviews Founder and CEO Jayne Hughes and molecular geneticist and specialist team member Prof. Donata Orioli on the aims, achievements, and activities of Amy and Friends.
期刊介绍:
FEBS Letters is one of the world''s leading journals in molecular biology and is renowned both for its quality of content and speed of production. Bringing together the most important developments in the molecular biosciences, FEBS Letters provides an international forum for Minireviews, Research Letters and Hypotheses that merit urgent publication.