Optimizing the CAS9 messenger RNA for vector-free gene editing

Sequence engineering and chemical modification of CAS9 mRNA

Cas9 Western Blot in CD34+ cells - Trilink poster "“Maximizing Translation of Cas9 mRNA Therapeutics by Sequence Engineering and Chemical Modification”"

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.

 


Subscribe to e-Newsletters on your favourite topics

Interested in learning more about tools like this?

Subscribe to thematic newsletters on your favourite research topics. 
Dimitri Szymczak, PhD
Written by Dimitri Szymczak, PhD
Dimitri Szymczak is a Technical Support Specialist and Product Manager at tebu-bio, and a fan of capoeira in his spare time.