Sequence engineering and chemical modification of CAS9 mRNA
Extract from poster “Maximizing Translation of Cas9 mRNA Therapeutics by Sequence Engineering and Chemical Modification”.
For transgenesis and therapeutic perspectives, vector-free delivery of the CAS9 for gene editing is highly preferable. It avoids the risk of hitting the genome due to the vector integration. Directly using CAS9 mRNA is a top option.
Furthermore, as a new promising class of therapeutic biologics, messenger RNA are the subject of numerous studies.
This year, Matthew Porteus’s and Trilink’s teams revealed key and practical results at the ASGC with a poster you can download below.
Notably, there is a large screen of the mRNA CAS9 modifications based on the indel formation. This interesting comparison includes also CAS9 RNP and reveals the high performance of the CleanCap U-depleted 5-moU CAS9 mRNA. We can also note that for the CleanCap CAS9 mRNA HPLC purification is not required.
“Maximizing Translation of Cas9 mRNA Therapeutics by Sequence Engineering and Chemical Modification”
Download your copy of the poster here.
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Gel filtration (GF), also referred as Size Exclusion Chromatography (SEC), plays a key role in the high quality purification of enzymes, polysaccharides, nucleic acids, proteins and other biological macromolecules. Gel filtration is the simplest and mildest of all chromatography techniques to separate biomolecules on the basis of difference in size. Nevertheless, the list of available columns is quite awesome and the characteristics of each of them are very different. Also, each characteristic highly influences the quality of the final purified product with important consequences in downstream applications.
