Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, Canada.
Department of Cell and Systems Biology, University of Toronto, Toronto, Canada.
Nucleic Acids Res. 2023 Jul 5;51(W1):W379-W386. doi: 10.1093/nar/gkad375.
MiniPromoters, or compact promoters, are short DNA sequences that can drive expression in specific cells and tissues. While broadly useful, they are of high relevance to gene therapy due to their role in enabling precise control of where a therapeutic gene will be expressed. Here, we present OnTarget (http://ontarget.cmmt.ubc.ca), a webserver that streamlines the MiniPromoter design process. Users only need to specify a gene of interest or custom genomic coordinates on which to focus the identification of promoters and enhancers, and can also provide relevant cell-type-specific genomic evidence (e.g. accessible chromatin regions, histone modifications, etc.). OnTarget combines the provided data with internal data to identify candidate promoters and enhancers and design MiniPromoters. To illustrate the utility of OnTarget, we designed and characterized two MiniPromoters targeting different cell populations relevant to Parkinson Disease.
微启动子，或紧凑启动子，是可以在特定细胞和组织中驱动表达的短 DNA 序列。虽然用途广泛，但由于它们在实现治疗基因表达位置的精确控制方面的作用，因此与基因治疗密切相关。在这里，我们介绍 OnTarget（http://ontarget.cmmt.ubc.ca），这是一个简化微启动子设计过程的网络服务器。用户只需指定感兴趣的基因或要关注启动子和增强子识别的自定义基因组坐标，还可以提供相关的细胞类型特异性基因组证据（例如可及染色质区域、组蛋白修饰等）。OnTarget 将提供的数据与内部数据相结合，以识别候选启动子和增强子并设计微启动子。为了说明 OnTarget 的实用性，我们设计并表征了两种针对与帕金森病相关的不同细胞群的微启动子。
