Cas9

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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CRISPR/Cas9 is relatively simple to implement, as the researcher fully controls the experimental design of the tools, from the sgRNA sequence to the Cas9 protein.

Let’s quickly look back to the basics… CRISPR Associated Protein 9 (Cas9) functions as part of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing system.

In this system, a single guide RNA (sgRNA) sequence targets a site of interest in the genome while the Cas9 protein performs a double stranded DNA cleavage at the targeted genomic location.� CRISPR/Cas9 system can be widely used to express or silence a gene, create mutations and variants… into the genome of the host cell.

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Optimizing the CAS9 messenger RNA for vector-free gene editing

Sequence engineering and chemical modification of CAS9 mRNA

Cas9 mRNA optimized for genome editing