deng245 发表于 2011-5-26 11:03 
据说RNAI技术是希望、但似乎石沉大海了
仍然有希望
Review
Nature Reviews Endocrinology, advance online publication, Published online 19 April 2011 | doi:10.1038/nrendo.2011.57 Subject Categories: Therapy (pharmacotherapy, radiotherapy, nutrition, alternative) | Diabetes (including the metabolic syndrome, hyperglycemia, hyperlipidemia, abdominal obesity, cardiovascular endocrinology) | Obesity | Metabolism RNAi-based therapeutic strategies for metabolic diseaseMichael P. Czech, Myriam Aouadi & Gregory J. Tesz About the authors Top of pageAbstractRNA interference (RNAi) is a robust gene silencing mechanism that degrades mRNAs complementary to the antisense strands of double-stranded, short interfering RNAs (siRNAs). As a therapeutic strategy, RNAi has an advantage over small-molecule drugs, as virtually all genes are susceptible to targeting by siRNA molecules. This advantage is, however, counterbalanced by the daunting challenge of achieving safe, effective delivery of oligonucleotides to specific tissues in vivo. Lipid-based carriers of siRNA therapeutics can now target the liver in metabolic diseases and are being assessed in clinical trials for the treatment of hypercholesterolemia. For this indication, a chemically modified oligonucleotide that targets endogenous small RNA modulators of gene expression (microRNAs) is also under investigation in clinical trials. Emerging 'self-delivery' siRNAs that are covalently linked to lipophilic moieties show promise for the future development of therapies. Besides the liver, inflammation of the adipose tissue in patients with obesity and type 2 diabetes mellitus may be an attractive target for siRNA therapeutics. Administration of siRNAs encapsulated within glucan microspheres can silence genes in inflammatory phagocytic cells, as can certain lipid-based carriers of siRNA. New technologies that combine siRNA molecules with antibodies or other targeting molecules also appear encouraging. Although still at an early stage, the emergence of RNAi-based therapeutics has the potential to markedly influence our clinical future.
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Author affiliationsM. P. Czech, M. Aouadi & G. J. Tesz
Program in Molecular Medicine, University of Massachusetts Medical School, 373 Plantation Street, Worcester, MA 01605, USA (M. P. Czech, M. Aouadi, G. J. Tesz). Correspondence to: M. P. Czech [email protected] Published online 19 April 2011
回顾
自然评论内分泌，网络版上公布，网上公布2011年4月19日|分类号：10.1038/nrendo.2011.57
学科门类：治疗（药物治疗，放射治疗，营养，替代）|糖尿病（包括代谢综合症，高血糖，高血脂，腹部肥胖，心血管内分泌学）|肥胖症|代谢
代谢疾病的RNAi为基础的治疗策略
迈克尔P.捷克，和Myriam Aouadi的Gregory J. Tesz及作者简介
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摘要
RNA干扰（RNAi）是一个强大的基因沉默机制，降低基因互补双链的反义链，短干扰RNA（siRNAs）。作为一种治疗策略，RNA干扰已比小分子药物的优势，因为几乎所有基因的siRNA分子容易被定位。这种优势是，然而，由于实现安全，有效地提供寡核苷酸在体内特定组织严峻的挑战平衡。 siRNA的治疗脂为基础的运营商现在可以针对肝脏代谢性疾病，并正在为高胆固醇血症治疗的临床试验评估。用于此适应症，一种化学修饰的寡核苷酸为目标内源性基因表达的小分子RNA（microRNA的）调制器的临床试验也在进行调查。新兴的自我交付'是亲油性基共价连接到siRNA的治疗显示了未来的发展前景。除了肝脏，在与肥胖和2型糖尿病患者脂肪组织的炎症可能是一个有吸引力的siRNA药物治疗的目标。葡聚糖微球包裹内政府可沉默基因的siRNAs炎性吞噬细胞，可以一定脂质为基础的siRNA的载体。新技术相结合的抗体或其他靶向分子的siRNA分子也出现令人鼓舞。虽然仍处于早期阶段，对RNAi为基础的疗法的出现有可能显着影响我们的临床前景。
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作者机构
体育捷克米，米Aouadi和克j的Tesz
计划在分子医学，马萨诸塞大学医学院，373人工林街，马萨诸塞州伍斯特市01605，美国（MP的捷克，米Aouadi，吉焦Tesz）。
通讯作者：MP的捷克michael.czech @ umassmed.edu
网上公布2011年4月19日
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楼主: 替比佩乐能
发表于 2011-5-26 19:24
