Development of new methods for controlling gene functions in vivo

该研究的目的是开发一种新的方法，用于以高空间和时间分辨率调节任何感兴趣基因的生物学功能。为了实现这一目标，我们一直在尝试合成笼状mRNA和DNA。我们设计了具有用于纯化、靶向和分子识别的亲和标签的笼状核苷酸的新前体分子。为了证明该化合物的实用性，我们开发了一种新的核苷酸锁定剂PNA-Bhc-diazo，其具有与靶基因互补的肽核酸（PNA）标签，用于序列选择性核苷酸锁定。设计并合成了一种可被可见光激活的新型笼状基团。

The purpose of the study is to develop a new method for regulating biological functions of any genes of interest with high spatial and temporal resolution. To this goal we have been trying to synthesize caged mRNAs and DNAs. We designed new precursor molecules of caged nucleotides having an affinity tag for purification, targeting and molecular recognition. To demonstrate utility of the compound, we developed a new nucleotide caging agent, PNA-Bhc-diazo, that has a peptide nucleic acid (PNA) tag complementary to a target gene for sequence-selective nucleotide caging. A new caging group which can be photo-activated by a visible light was designed and synthesized.