Daley George Q, Goodell Margaret A, Snyder Evan Y
Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Children's Hospital, Cambridge, MA 02142, USA.
Hematology Am Soc Hematol Educ Program. 2003:398-418. doi: 10.1182/asheducation-2003.1.398.
Studies of the regenerating hematopoietic system have led to the definition of many of the fundamental principles of stem cell biology. Therapies based on a range of tissue stem cells have been widely touted as a new treatment modality, presaging an emerging new specialty called regenerative medicine that promises to harness stem cells from embryonic and somatic sources to provide replacement cell therapies for genetic, malignant, and degenerative conditions. Insights borne from stem cell biology also portend development of protein and small molecule therapeutics that act on endogenous stem cells to promote repair and regeneration. Much of the newfound enthusiasm for regenerative medicine stems from the hope that advances in the laboratory will be followed soon thereafter by breakthrough treatments in the clinic. But how does one sort through the hype to judge the true promise? Are stem cell biologists and the media building expectations that cannot be met? Which diseases can be treated, and when can we expect success? In this review, we outline the realms of investigation that are capturing the most attention, and consider the current state of scientific understanding and controversy regarding the properties of embryonic and somatic (adult) stem cells. Our objective is to provide a framework for appreciating the promise while at the same time understanding the challenges behind translating fundamental stem cell biology into novel clinical therapies.
对再生造血系统的研究已促成了许多干细胞生物学基本原理的定义。基于一系列组织干细胞的疗法已被广泛吹捧为一种新的治疗方式,预示着一个名为再生医学的新兴专业的出现,该专业有望利用来自胚胎和体细胞来源的干细胞为遗传、恶性和退行性疾病提供替代细胞疗法。干细胞生物学带来的见解还预示着作用于内源性干细胞以促进修复和再生的蛋白质和小分子疗法的发展。再生医学新出现的诸多热情很大程度上源于人们希望实验室的进展能很快在临床上带来突破性治疗。但如何在炒作中辨别以判断其真正前景呢?干细胞生物学家和媒体是否在构建无法实现的期望?哪些疾病可以得到治疗,我们何时能期待成功?在本综述中,我们概述了最受关注的研究领域,并思考了关于胚胎干细胞和体细胞(成体)干细胞特性的当前科学理解状况及争议。我们的目标是提供一个框架,既能认识到其前景,同时又能理解将基础干细胞生物学转化为新型临床疗法背后的挑战。