CRISPR or Clustered Regularly Interspaced Short Palindromic Repeats is a simple yet powerful gene-editing technology that enables the precise and permanent modification of DNA in targeted cells and tissues. The components of the CRISPR editing system can be encoded into RNA molecules and delivered to organs to effectively edit the genome. In this sense, CRISPR machinery can be delivered to its destination much like other RNA technologies, and as such, opens doors for therapeutic intervention. Successful clinical application of messenger RNA (mRNA) delivered in lipid nanoparticles (LNPs), as seen with the Covid19 mRNA vaccinations, has spawned a major interest in utilising these technologies for a wider variety of therapeutic modalities.
Our CRISPR Validation platform harnesses both RNA therapeutics and LNP technologies to facilitate CRISPR gene editing in preclinical models, with a focus towards the development of novel treatments for cardiometabolic disease.
Our capabilities
- Formulation and optimisation of lipid nanoparticles using microfluidic mixing devices (NanoAssemblr Ignite and Spark) or benchtop methods (vortex and pipetting).
- Screening of CRISPR guides in cell or in vivo systems.
- Ability to deliver, visualise, quantify and assess biodistribution of lipid nanoparticles in vivo.
- Access to tissue-specific Cas9-Cre transgenic preclinical models (liver, adipose tissue, heart and muscle).
- Expertise in preclinical metabolic phenotyping.
- Establishment of models using one-time injections in vivo to develop disease models, including models of dyslipidemia (LDLR-KO, ApoE-KO, PCSK9i) with broad applications for therapeutic testing.
