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分子与细胞疗法:新挑战与新机遇

Molecular and Cellular Therapies: New challenges and opportunities.

作者信息

Wang Xiangdong, Peer Dan, Petersen Bryon

机构信息

Shanghai Institute of Respiratory Diseases; Department of Respiratory Medicine, Fudan University Zhongshan Hospital, Shanghai, Sweden ; Institute of Clinical Science, Lund University, Lund, Sweden.

Department of Cell Research & Immunology, Laboratory of NanoMedicine, and the center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel.

出版信息

Mol Cell Ther. 2013 Nov 6;1:1. doi: 10.1186/2052-8426-1-1. eCollection 2013.

Abstract

Gene therapy is suggested to be one of the most specific and efficient modulations for gene deficient diseases and extended to other diseases like cancer and inflammation, even though there are still challenges to be faced, such as specific and selective delivery, minimal to no toxicity, efficient metabolism, simplicity, and measurable efficiency. It is important to identify and validate drug-able disease-specific targets for molecular and cellular therapies, while it is equally important to have disease biomarkers to trace and define the biological effects of molecular and cellular therapies. The importance and significance of allostery in molecular and cellular therapies and "allosteric disease", "allosteric effect", and "allosteric drug" should be more carefully examined and validated. Cell therapy has been attracting an increasing amount of consideration in the development of new treatments for diseases. Molecular and Cellular Therapies (MCT) is a new, open-access journal, devoted to molecular mechanisms, preclinical and clinical research and development of gene-, peptide-, protein-, and cell-based therapies.

摘要

基因治疗被认为是针对基因缺陷疾病最具特异性和高效性的调控方法之一,并已扩展到癌症和炎症等其他疾病,尽管仍面临一些挑战,如特异性和选择性递送、极低或无毒性、高效代谢、操作简便以及可测量的疗效。识别和验证用于分子和细胞治疗的可成药疾病特异性靶点很重要,同时拥有疾病生物标志物以追踪和定义分子和细胞治疗的生物学效应同样重要。变构在分子和细胞治疗中的重要性和意义,以及“变构疾病”“变构效应”和“变构药物”应得到更仔细的研究和验证。细胞治疗在疾病新疗法的开发中越来越受到关注。《分子与细胞治疗》(MCT)是一本新的开放获取期刊,致力于基因、肽、蛋白质和细胞疗法的分子机制、临床前和临床研究与开发。

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