PDF(Pubmed)
Abstract：
Aptamers are RNA/DNA oligonucleotide molecules that specifically bind to a targeted complementary molecule. As potential recognition elements with promising diagnostic and therapeutic applications, aptamers, such as monoclonal antibodies, could provide many treatment and diagnostic options for blood diseases. Aptamers present several superior features over antibodies, including a simple in vitro selection and production, ease of modification and conjugation, high stability, and low immunogenicity. Emerging as promising alternatives to antibodies, aptamers could overcome the present limitations of monoclonal antibody therapy to provide novel diagnostic, therapeutic, and preventive treatments for blood diseases. Researchers in several biomedical areas, such as biomarker detection, diagnosis, imaging, and targeted therapy, have widely investigated aptamers, and several aptamers have been developed over the past two decades. One of these is the pegaptanib sodium injection, an aptamer-based therapeutic that functions as an anti-angiogenic medicine, and it is the first aptamer approved by the U.S. Food and Drug Administration (FDA) for therapeutic use. Several other aptamers are now in clinical trials. In this review, we highlight the current state of aptamers in the clinical trial program and introduce some promising aptamers currently in pre-clinical development for blood diseases.
摘要：
适体是特异性结合靶向互补分子的RNA/DNA寡核苷酸分子。作为具有有前途的诊断和治疗应用的潜在识别元素，适体,如单克隆抗体，可以为血液疾病提供许多治疗和诊断选择。适体呈现出优于抗体的几个特征，包括简单的体外选择和生产，易于修饰和共轭，高稳定性,低免疫原性。作为抗体的有希望的替代品，适体可以克服目前单克隆抗体治疗的局限性，提供新的诊断，治疗性的,和血液疾病的预防性治疗。几个生物医学领域的研究人员，如生物标志物检测，诊断,成像,和靶向治疗,已经广泛研究了适体，在过去的二十年中，已经开发了几种适体。其中之一是pegaptanib钠注射液，一种基于适体的治疗剂，起到抗血管生成药物的作用，它是第一个由美国食品和药物管理局（FDA）批准用于治疗用途的适体。其他几种适体现在正在临床试验中。在这次审查中,我们重点介绍了临床试验计划中适体的现状，并介绍了一些目前正在血液病临床前开发中的有前途的适体。
