Introduction: Gene Therapy and Gene Transfer Projects of the 7th Framework Programme for Research and Technological Development of the European Union (Second Part).
Introduction: Gene Therapy and Gene Transfer Projects of the 7th Framework Programme for Research and Technological Development of the European Union (Second Part).
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简介：欧盟第七期研究与技术发展框架计划基因治疗和基因转移项目（第二部分）。
DOI：
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发表时间：
2015
影响因子：
--
通讯作者：
R. Draghia‐Akli
中科院分区：
文献类型：
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作者：
David Gancberg;A. Hoeveler;R. Draghia‐Akli
以下是欧盟在第七届研究与创新框架计划（2007-2013）期间资助的12个正在进行的基因治疗/基因转移项目的第二个作品。这些项目中有一半涉及罕见疾病（EuroFancolen，MeuSIX，Net 4CGD，Scope-DMD，Skip-NMD，SUPERSIST，Thalamoss），而其他研究常见疾病，如癌症（ATECT，ATTACK，SUPERSIST，MERIT），年龄相关性黄斑变性（TargetAMD）或血管疾病（EVERREST）。这些国际合作项目中的大多数将进行临床试验，表明该领域现在已经成熟，可以转向床边。每个项目的介绍都包括主要的研究成果和出版物，此外，还提供了专门的网站，在那里可以找到有关该联盟结构和成就的详细信息。基因治疗试验和基因工程技术现在经常在科学新闻中突出显示。嵌合抗原受体（CAR）T细胞技术治疗CD 19 + B细胞白血病的惊人结果和新一代病毒载体的安全性证据，导致该领域的专家在欧洲基因和细胞治疗学会的最后一届年会上提出基因治疗作为某些罕见疾病的一线治疗。此外，国际罕见病研究联盟（IRDiRC）已经为罕见病提供了140多种新疗法，到2020年的目标是200种。基因和细胞技术比以往任何时候都更加互补，非病毒载体的发展作为基因工程的特定工具和诱导多能干细胞系的产生作为再生医学的潜在候选人。创新药物倡议是欧盟和欧洲制药工业和协会联合会之间的公私合作伙伴关系，正在通过两个项目StemBANCC和EBiSC支持这些细胞系的生成，表征和库。制药业在新的基因和细胞技术领域的投资不断增加，表明该领域进行人体试验的时机已经成熟。
We present hereafter the second opus of 12 ongoing gene therapy/gene transfer projects funded by the European Union during the 7th Framework Programme for Research and Innovation (2007–2013). Half of these projects address rare diseases (EuroFancolen, MeuSIX, Net4CGD, Scope-DMD, Skip-NMD, SUPERSIST, Thalamoss), while others investigate common disorders such as cancer (ATECT, ATTACK, SUPERSIST, MERIT), age-related macular degeneration (TargetAMD), or vascular disorders (EVERREST). Most of these international collaborative projects will perform clinical trials, demonstrating that the field is now ripe for moving to the bedside. Each project presentation includes main findings and publications in addition to their dedicated website where details about consortium’s structure and achievements can be found. Gene therapy trials and genetic engineering technologies are now regularly highlighted in the scientific news. The spectacular results obtained with the chimeric antigen receptor (CAR) T cell technology for the treatment of CD19 + B cell leukaemias and the safety evidence of new generation of viral vectors led the experts in the field to propose gene therapy as first-line treatment for some rare diseases during the last annual conference of the European Society for Gene and Cell Therapy. Moreover, the dynamism for search of innovative therapeutic modalities is exemplified within the International Rare Diseases Research Consortium (IRDiRC) that has already now delivered more than 140 new therapies for rare diseases, on the target objective of 200 by 2020. Gene and cell technologies are more complementary than ever with the development of nonviral vectors as specific tools for genetic engineering and the generation of induced pluripotent stem cell lines as potential candidates in regenerative medicine. The Innovative Medicine Initiative, a public–private partnership between the European Union and the European Federation of Pharmaceutical Industries and Associations, is supporting the generation, the characterization, and the banking of those cell lines via two projects, StemBANCC and EBiSC. The growing investments of the pharmaceutical industry in the area of new gene and cell technologies suggest that the field is now ripe for human trials.
利用工程化 T 细胞迈向合成生物学：漫长的旅程才刚刚开始。
影响因子：
4.2
作者：
June,CarlH
通讯作者：
June,CarlH