Vertically integrated translational studies of PDX1 as a therapeutic target for pancreatic cancer via a novel bifunctional RNAi platform.
Vertically integrated translational studies of PDX1 as a therapeutic target for pancreatic cancer via a novel bifunctional RNAi platform.
复制标题
通过新型双功能 RNAi 平台对 PDX1 作为胰腺癌治疗靶点进行垂直整合转化研究。
DOI：
10.1038/cgt.2013.84
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发表时间：
2014
影响因子：
6.4
通讯作者：
Brunicardi,FC
中科院分区：
文献类型：
--
作者：
Wu,J;Liu,S;Yu,J;Zhou,G;Rao,D;Jay,CM;Kumar,P;Sanchez,R;Templeton,N;Senzer,N;Maples,P;Nemunaitis,J;Brunicardi,FC
RNA干扰（RNAi）是一种强大的科学研究新工具，也是一种有前途的靶向基因治疗新形式，目前已应用于临床试验。双功能短发夹RNA （shRNA）是一种人工合成的RNAi分子，利用多种内源性RNAi途径特异性沉默靶基因。胰腺和十二指肠同源盒1 （PDX1）是胰腺发育、β细胞分化、正常β细胞功能和胰腺癌的关键调节因子。我们的目的是回顾确定PDX1作为胰腺癌特异性的潜在RNAi靶点的过程，以及潜在的机制和各种形式的RNAi，以及随后的PDX1靶向双功能shRNA治疗的测试和开发。
RNA interference (RNAi) represents a powerful, new tool for scientific investigation as well as a promising new form of targeted gene therapy, with applications currently in clinical trials. Bifunctional short hairpin RNA (shRNA) are synthetic RNAi molecules, engineered to utilize multiple endogenous RNAi pathways to specifically silence target genes. Pancreatic and duodenal homeobox 1 (PDX1) is a key regulator of pancreatic development, β-cell differentiation, normal β-cell function and pancreatic cancer. Our aim is to review the process of identifying PDX1 as a specific, potential RNAi target in pancreatic cancer, as well as the underlying mechanisms and various forms of RNAi, with subsequent testing and development of PDX1-targeted bifunctional shRNA therapy.