Macrocyclic Library with Conformationally Diverse Scaffold: Synthesis and Biology
Poster Oct 21, 2005
N. Qvit, S. Gazal, H. Reuveni, M. Niv, G. Blum, A. Feldstein, S. Meushar, A. Friedler and C. Gilon
Here we present the SIB concept, based on macrocyclic libraries in which the ring size is varied in gradual manner (conformational diversity). This allows selection of bioactive molecules based on their conformational complementary.
We utilized paired synthetic crRNAs coupled with our synthetic tracrRNA in cells transduced with lentiviral Cas9 to perform a functional knockout on hsa-miR-221. This three-part system (crRNA, tracrRNA and Cas9) has demonstrated efficient gene editing when used with only one guide RNA, but the goal was to use two crRNAs to remove the entire stem-loop.READ MORE