Therapeutics Core (Page 286)

PR0JECT摘要(请参阅说明)： 治疗核心的目标是为寻求纳米配方的逆转录病毒和神经保护疗法的研究人员提供纳米制剂，以增加中枢神经系统(CNS)的外显率。开发的配方解决了单核细胞和其他免疫细胞如何被利用来传递药物的问题。在这些新疗法可以用于人类之前，我们将在经过充分验证的实验室和动物模型中确定最佳剂量和配方管理。核心面临的问题的症结，即纳米配方抗逆转录病毒疗法能否显示持续的抗逆转录病毒反应和药物在组织中的缓慢释放，现在已经得到解决。该核心解决了艾滋病毒和神经艾滋病治疗中的一个具体和重要的问题，对其他神经退行性疾病的治疗干预具有更广泛的影响。通过这个项目，我们将识别和制造目前使用的有效抗逆转录病毒药物的候选纳米制剂。这些将测试人类疾病的模型系统，从培养的单核细胞到小鼠再到猴子，以检查药代动力学、安全性和有效性。总体而言，这一核心代表了可能被证明是长效治疗剂开发方面的重大进步，这种长效治疗剂可以导致对全身和中枢神经系统人类疾病的真正治疗。

PR0JECT SUMMARY (See instructions): The goals of the Therapeutics core are to provide nanoformulations to invesfigators pursuing nanoformulated ntiretroviral and neuroprotective therapies for increased central nervous system (CNS) penetrance. The formulations developed address how monocytes and other immune cells may be harnessed for drug delivery. Before such novel therapies can be administered to people, we will determine, in well-validated laboratory and animal models, the optimal doses and formulation administration. The crux of the problem facing the Core, namely can nanoformulated antiretroviral therapy show sustained antiretroviral responses and slow release of drug in tissues, has now been addressed. The Core addresses a specific and important issue in the treatment of HIV and neuroAIDS, with broader implications for therapeutic interventions to other neurodegenerative diseases. Through this project, we will identify and manufacture candidate Nanoformulations of currently used efficacious antiretrovirals. These will be tested model systems of human disease, ranging from cultured monocytes to mice to monkeys, to examine pharmacokinefic, safety and efficacy. This Core, overall, represents work that may prove to be a major advance in the development of long-lasting therapeutic agents that can lead to real treatments both systemic and CNS human disease.