CRISPR-Cas-Mediated Gene Editing

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The first genome editing technologies were developed in the late 1900s. More recently, a new genome editing tool called CRISPR, invented in 2009, has made it easier than ever to edit DNA. CRISPR is simpler, faster, cheaper, and more accurate than older genome editing methods.

CRISPR-based technologies are techniques that enable to modify DNA in the genome, therefore, they can be used to correct defective genes. These technologies act like scissors, cutting the DNA at a specific spot. Then scientists can remove, add, or replace the DNA where it was cut.

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Editing the genomes of yeast, bacteria, mice, zebrafish, and other organisms that scientists commonly study has led to countless discoveries about how the genome is connected to physical traits. Scientists have had the knowledge and ability to edit genomes for many years, but CRISPR technology has brought major improvements to the speed, cost, accuracy, and efficiency of gene editing.

 

CRISPR-Cas9 is a gene editing technology that researchers are harnessing to explore two approaches for reducing the symptoms of disease. Our approach will correct the DMD mutation by removing mutated exons to restore a shorter but functional dystrophin, and the second approach will edit the DNA bases for personalized therapy to restore functional dystrophin production. At Art Bioscience we are committed to finding a treatment or a cure for children with Duchenne, and we are developing both approaches in parallel.

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Genome editing holds both tremendous therapeutic promise and significant potential risk. We believe that with our LNPs delivery system we are reducing that risk. Cas9 mRNA encapsulated in LNPs delivery system penetrates the cell Cytoplasm, the mRNA is translated to Cas9 protein, then breaks down and doesn’t stay in the body, which means that repeated injections will be required. This will enable us to develop personalized gene editing therapies. Patient dosing can be tailored to his / her specific needs based on disease progression and tolerability.