A Robust High Throughput Physicochemical Screen for Phospholipidosis
Poster May 09, 2007
Phospholipidosis (PLD), accumulation of phospholipids in tissues such as lungs, kidneys and liver, has gained increasing attention, particularly concerning drugs intended for long term usage. The poster presents a simple HT physicochemical screen, predicting PLD in real-time by surface activity profiling (using Kibron Delta-8 analyzer). The results are equal or better than derived from a cell based assay, throughput is up 450 compounds/24 hrs and drug consumption < 1 mg/compound.
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