{"title":"Demystifying gene(tic) therapies","authors":"Chun-Hung Chan , David A. Pearce","doi":"10.1016/j.ejmg.2024.104963","DOIUrl":null,"url":null,"abstract":"<div><p>This article summarizes the discussion from a session entitled “Demystifying gene therapies” that was held at the joint RE(ACT) congress and IRDiRC conference, 14–15 March 2023 in Berlin, Germany. The focus of this session was to discuss the changing landscape of genetic therapies and whether current resources exist to provide adequate education to stakeholders, such as researchers, clinicians, patient advocates, legislators, as well as the patients and their families. The goal of this article is not to provide a comprehensive overview of the current landscape in genetic therapies, but rather to highlight resources that may be useful to help “demystify” the myriad of genetic therapeutic approaches.</p></div>","PeriodicalId":11916,"journal":{"name":"European journal of medical genetics","volume":"71 ","pages":"Article 104963"},"PeriodicalIF":1.6000,"publicationDate":"2024-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1769721224000557/pdfft?md5=03051b5d5212d03cc724e72f838a6890&pid=1-s2.0-S1769721224000557-main.pdf","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"European journal of medical genetics","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1769721224000557","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0
Abstract
This article summarizes the discussion from a session entitled “Demystifying gene therapies” that was held at the joint RE(ACT) congress and IRDiRC conference, 14–15 March 2023 in Berlin, Germany. The focus of this session was to discuss the changing landscape of genetic therapies and whether current resources exist to provide adequate education to stakeholders, such as researchers, clinicians, patient advocates, legislators, as well as the patients and their families. The goal of this article is not to provide a comprehensive overview of the current landscape in genetic therapies, but rather to highlight resources that may be useful to help “demystify” the myriad of genetic therapeutic approaches.
期刊介绍：
The European Journal of Medical Genetics (EJMG) is a peer-reviewed journal that publishes articles in English on various aspects of human and medical genetics and of the genetics of experimental models.
Original clinical and experimental research articles, short clinical reports, review articles and letters to the editor are welcome on topics such as :
• Dysmorphology and syndrome delineation
• Molecular genetics and molecular cytogenetics of inherited disorders
• Clinical applications of genomics and nextgen sequencing technologies
• Syndromal cancer genetics
• Behavioral genetics
• Community genetics
• Fetal pathology and prenatal diagnosis
• Genetic counseling.
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