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  • OVERVIEW
Therapeutic Genome Editing mRNA Development
  • OVERVIEW

Therapeutic Genome Editing mRNA Development

Gene correction of transplantable cells using CRISPR/Cas9-based tools is a realistic scenario for autologous cell replacement therapies to restore organ function in many genetic disorders.  However, the application of this technology has been limited by the technical challenge of achieving safe, effective, and specific in vivo delivery of the CRISPR-Cas9 genome editing components.

Our Service Portfolio


1. mRNA design


2. In Vivo Screening of LNPs for mRNA Delivery


3. Optimization of the Formulation


4. LNP-formulated mRNA preparation


5. In vitro mRNA validation


6. NGS Sequencing Analysis


7. In Vivo Cas9 mRNA/sgLoxP Delivery


8. Off-target analysis


Advantages of our technology


1. mRNA based gene editing tools is an alternative to DNA-based gene editing technologies. One advantage lies in the fact that mRNA’s effects are temporary, in contrast to the permanent effect of DNA. Moreover, mRNA carries no risk of genomic integration, which might not just be a theoretical risk for DNA. This gives mRNA an inherent safety advantage over DNA-based therapeutics.


2. mRNA-based gene have significantly enhanced translational efficience of foreign mRNA in host cells, mainly owe to the discovery of 5′mRNA anti-reverse cap analogs (ARCA), the insertion of additional untranslated regions, and poly(A) tails.


3. The immunogenic reaction of toll-like receptor-activated mRNA is weaker than the unmethylated CpG motifs of DNA recognized by TLR9.


4. The mRNA transfection into host cells can be much easier because its construct is far smaller than pDNA. Furthermore, mRNA gene therapy circumvents the need for selecting a specific promoter, and thus the transfection process is relatively efficient and facile.


5. Protein translation takes place almost immediately after mRNA transfection because of it's functionality in the cytoplasm without the need to enter into the nucleus.


Our client relations specialists will handle your request seamlessly from a sequence, straight through to deliverable material produced to your specifications. Seattle Genova’s state-of-the-art quality systems ensure your material will meet your specifications.

Therapeutic Genome Editing mRNA Development

Gene correction of transplantable cells using CRISPR/Cas9-based tools is a realistic scenario for autologous cell replacement therapies to restore organ function in many genetic disorders.  However, the application of this technology has been limited by the technical challenge of achieving safe, effective, and specific in vivo delivery of the CRISPR-Cas9 genome editing components.

Our Service Portfolio


1. mRNA design


2. In Vivo Screening of LNPs for mRNA Delivery


3. Optimization of the Formulation


4. LNP-formulated mRNA preparation


5. In vitro mRNA validation


6. NGS Sequencing Analysis


7. In Vivo Cas9 mRNA/sgLoxP Delivery


8. Off-target analysis


Advantages of our technology


1. mRNA based gene editing tools is an alternative to DNA-based gene editing technologies. One advantage lies in the fact that mRNA’s effects are temporary, in contrast to the permanent effect of DNA. Moreover, mRNA carries no risk of genomic integration, which might not just be a theoretical risk for DNA. This gives mRNA an inherent safety advantage over DNA-based therapeutics.


2. mRNA-based gene have significantly enhanced translational efficience of foreign mRNA in host cells, mainly owe to the discovery of 5′mRNA anti-reverse cap analogs (ARCA), the insertion of additional untranslated regions, and poly(A) tails.


3. The immunogenic reaction of toll-like receptor-activated mRNA is weaker than the unmethylated CpG motifs of DNA recognized by TLR9.


4. The mRNA transfection into host cells can be much easier because its construct is far smaller than pDNA. Furthermore, mRNA gene therapy circumvents the need for selecting a specific promoter, and thus the transfection process is relatively efficient and facile.


5. Protein translation takes place almost immediately after mRNA transfection because of it's functionality in the cytoplasm without the need to enter into the nucleus.


Our client relations specialists will handle your request seamlessly from a sequence, straight through to deliverable material produced to your specifications. Seattle Genova’s state-of-the-art quality systems ensure your material will meet your specifications.

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