In‐vivo synthesis of GM1 Ganglioside through CRISPR‐Cas9 mutation in CHO‐K1, Chinese hamster ovary cells (Cricetulus griseus)

GM1 ganglioside has been shown to have therapeutic properties against, Parkinson’s disease, Alzheimer’s, dementia and with dramatic results in Huntington’s Disease (Holler, et al. , 2016). Studies also show recovery in CNS related injuries with GM1 therapy (Karpiak et al. , 1986). New studies continue to confirm GM1 gangliosides therapeutic properties and may increase the need for GM1 ganglioside. Currently, large scale, synthesis of this ganglioside involves isolation from a natural product. New synthesis methods, such as chemical synthesis, yields an insignificant quantity for alternative large‐scale production (Aureli et al. , 2015). Research presented here attempts to address a new method of large‐scale production by in vivo synthesis of GM1 ganglioside through a CRISPR‐Cas9 induced mutation in Chinese Hamster Ovary cells (CHO‐K1) by introducing the mutation of the GLB1 gene that causes a substantial accumulation of GM1 in cells known as GM1 gangliosidosis. In‐vivo synthesis provides relief from natural product isolation and may be cost effective to chloroform: methanol protocols while providing similar results. Support or Funding Information This material is based upon work supported by the National Science Foundation/EPSCoR Cooperative Agreement #OIA‐1849206 and by the State of South Dakota, SD‐GOED, NSF I‐Corps Team program and Dr. Larry and Sue Holler, GlycoScience Research Incorporated