Fundamental and translational research in Cystic Fibrosis - why we still need it.
Fundamental and translational research in Cystic Fibrosis - why we still need it.
复制标题
囊性纤维化的基础和转化研究 - 为什么我们仍然需要它。
DOI：
10.1016/j.jcf.2022.12.010
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发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Pedemonte,Nicoletta
中科院分区：
文献类型：
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作者：
Farinha,CarlosM;Brodsky,JeffreyL;Pedemonte,Nicoletta
囊性纤维化（CF）的临床治疗在过去十年中经历了重大变化，因为靶向CFTR蛋白中的基本缺陷的疗法被批准。在其他几个领域也取得了重大的科学进展，这些领域可能在未来导致新的治疗方法，可以帮助所有患有这种疾病的人对抗CF。因此，专注于CF领域的基础研究已经并将继续具有重要意义。这是欧洲囊性纤维化协会（ECFS）的目标之一，自2004年以来，ECFS每年都举办基础科学会议（BSC）。这期特刊涵盖了2022年3月在葡萄牙阿尔布费拉举行的第17届ECFS BSC上的主题和讨论，并强调了在理解CFTR、使用个性化药物以及开发创新策略以确定突破性疗法方面的进展。这篇介绍突出了整个特刊中提出的主题，从而强调了CF基础研究的相关性。
Clinical treatments for cystic fibrosis (CF) underwent significant changes in the last decade as therapies targeting the basic defect in the CFTR protein were approved. Significant scientific progress has also been made in several other areas that may lead in the future to novel therapeutic approaches that can help fight CF in all individuals living with this disease. Thus, focusing on fundamental research in the CF field has and will continue to be of great importance. This has been one of the aims of the European Cystic Fibrosis Society (ECFS), which has promoted the ECFS Basic Science Conference (BSC) every year since 2004. This special issue covers the topics featured and discussed at the 17thECFS BSC, held in Albufeira (Portugal) in March 2022, and highlights advances in understanding CFTR, in using personalized medicine, and in developing innovative strategies to identify breakthrough therapies. This introduction highlights the topics presented throughout this special issue, thereby underscoring the relevance of fundamental research in CF.