How genetic editing targets specific areas…

Deep Genomics is developing an artificial intelligence (AI)-enhanced approach to genetic editing. Ultimately, Deep Genomics’ work could one day compete with CRISPR technology. And its approach is great for diseases that occur when a genetic mutation causes certain cells to produce the wrong proteins. 

Deep Genomics can correct this simply by “turning off” those cells.  Of course, specificity is key with gene editing since we don’t want any off-target changes. The good news is, Deep Genomics can target specific areas of our DNA for treatment.  This happens through “steric blocking oligonucleotides.” 

These are short stretches of DNA or RNA that attach to specific places in the patient’s RNA. With genetic editing technologies using RNA, the therapy can be steered to precise locations. To use a loose analogy, it’s like two unique pieces of a puzzle. The therapy is only delivered to the precise location where the RNA matches. 

 The therapy can then modify the way the cells process the RNA and create proteins. No permanent changes happen to the DNA. Here’s an illustration of the process:

As you can see from the left and right sides of the graphic, the DNA itself hasn’t changed. The genetic editing procedure just affects the protein output. And because of how it’s “coded,” the treatment clearly targets the intended genes.  Off-target editing was a much larger concern five to seven years ago, when genetic editing technology was more of a theory. Today, it has already been proven to cure diseases like beta thalassemia and sickle cell disease, with many others in clinical trials now.  

All these genetic editing therapies must go through the normal clinical trial process as defined by the U.S. Food and Drug Administration (FDA). And Phase 1 is specifically designed to determine safety and inform decisions regarding dosing levels for the Phase 2 portion of the trials.   While I don’t expect many problems to be found, any issues with off-target editing will likely be discovered in the laboratory, even before a therapy advances to Phase 1 clinical trials. 

What makes genetic editing technology so exciting is that it is the definition of precision medicine. It’s designed to be precise and go straight to its desired target instead of a therapy that affects the entire body like chemotherapy. So we won’t have to worry about turning any organs off when it comes to genetic editing.