School of Science Division of Life Science 23 Molecular Regulation of Axon Regeneration Supervisor: LIU Kai / LIFS Student: ZHAO Bolin / BCB Course: UROP 1100, Summer In recent research, the spontaneous immune signaling pathway cyclic GMP–AMP synthase (cGAS) –2 3 -cyclic GMP–AMP (cGAMP)–stimulator of interferon genes (STING) pathway has been discovered. This signaling pathway plays a crucial role in cellular life activities and neural cell regeneration, and is associated with numerous human diseases. One important intermediate substance in this signaling pathway, cGAMP, is critical both intracellularly and intercellularly, eliciting various downstream reactions. Unlike cAMP and cGMP, only one mammalian hydrolase for cGAMP has been identified: ectonucleotide pyrophosphatase phosphodiesterase 1 (ENPP1). This secreted protein is significantly present on the plasma and membrane surfaces. Its extracellular presence leads to the degradation of cGAMP, thereby influencing its function. Therefore, research on ENPP1 inhibitors is crucial. The screening of ENPP1 inhibitors requires STING sensor that detects the presence of cGAMP. This report will present findings on the effects of a transfected STING sensor in HEK293T cells. Additionally, dorsal root ganglion (DRG) cells from mice are commonly used materials in neuroscience research; this report will also discuss nocodazole's impact on axon regeneration in isolated DRG cells. Furthermore, this report will cover my learning progress as well as outstanding issues that require resolution. Genome Editing by CRISPR Supervisor: LIU Zhen / LIFS Student: JIA Elena Minghua / BCB Course: UROP 1100, Summer Ciliopathies are a group of genetic disorders caused by defects in the structure or function of cilia, leading to a wide range of clinical manifestations, including respiratory, renal, and reproductive abnormalities. Despite significant advances, the genetic and molecular mechanisms underlying ciliopathies remain incompletely understood. This study employs CRISPR-Cas9 gene editing to investigate the roles of specific genes concerning ciliopathies, particularly those involved with the respiratory system. By creating knockout models, we aimed to elucidate the molecular mechanisms underlying these disorders. Our findings provide insights into the genetic basis of ciliary function and offer potential targets for therapeutic development in ciliopathies.
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