Baby STEPS: a giant leap for cell therapy in neonatal brain injury.
Baby STEPS: a giant leap for cell therapy in neonatal brain injury.
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DOI：
10.1203/pdr.0b013e31821d0d00
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发表时间：
2011-07
影响因子：
3.6
通讯作者：
Weiss MD
中科院分区：
文献类型：
--
作者：
Borlongan CV;Weiss MD
我们推进婴儿STEPS或干细胞治疗作为中风的新兴范例，作为促进实验室对新生儿脑病细胞治疗的安全性和有效性进行关键评估的指南。需要仔细考虑动物模型在模拟人类新生儿脑损伤中的临床相关性，选择最佳干细胞供体，以及在小型和大型动物模型中应用功能结果测定，这些都是临床前工作和开始了解这种细胞治疗机制的基础。临床前研究将有助于我们制定严格的人体临床试验，其中不仅包括疗效测试，还包括安全性指标监测和作用机制证明。这个模式构成了Baby Steps的基础。我们的目标是响应紧急呼吁，加强细胞治疗从实验室到临床的成功转化。
We advance Baby STEPS or Stem cell Therapeutics as an Emerging Paradigm for Stroke as a guide in facilitating the critical evaluation in the laboratory of the safety and efficacy of cell therapy for neonatal encephalopathy. The need to carefully consider the clinical relevance of the animal models in mimicking human neonatal brain injury, selection of the optimal stem cell donor, and the application of functional outcome assays in small and large animal models serve as the foundation for preclinical work and beginning to understand the mechanism of this cellular therapy. The preclinical studies will aid our formulation of a rigorous human clinical trial which encompasses not only efficacy testing but also monitoring of safety indices and demonstration of mechanisms of action. This schema forms the basis of Baby STEPS. Our goal is to resonate the urgent call to enhance the successful translation of cell therapy from the laboratory to the clinic.