Medical research capacity to isolate genetic mutations responsible for disease have been revolutionized through the innovation of CRISPR technology.

Comprising an expanding set of tools, the technology behind CRISPR manipulates individual genes (and gene expression) by isolating DNA with Cas9 and Cas12 enzymes and isolating RNA with the Cas13 enzyme. A scientific breakthrough that offers multiple frameworks for solving mutations. For example, targeting short-lived RNA disease mutations results in no permanent genome alterations. 

RESCUE which is short for “RNA Editing for Specific to C to U Exchange” is one such new strategy researchers have invented by utilizing deactivated Cas13 in leading RESCUE to isolated base levels of cytosine on RNA imprints. Excess cytosine is transformed into uridine through advanced enzyme programming which initiates new RNA instructions that direct the transformation.  

As recently profiled in the journal Science, RESCUE’s expansion of CRISPR technology offers meaningful tools in isolating modification positions of proteins. The ramifications of protein activity of phosphorylation areas are regularly found in cancer pathways. These areas are proverbial “engage/disengage” molecules found in protein activity that can be manipulated to treat bodily changes causing disease.

RESCUE isolation expands the capability of modifying the functional activity of several proteins like methylation, phosphorylation and glycosylation through editing. “By developing this new enzyme and combining it with the programmability and precision of CRISPR, we were able to fill a critical gap…,” says Dr. Zhang, Professor of Neuroscience at MIT. 

DNA level editing results in permanent changes but RNA editing holds great advantages through reversibility. RESCUE can be employed in temporary modifications such as editing human cells yielding a temporary increase in cell growth. The temporal nature of RESCUE offers real advantages to controlling cell growth and cancer. Further, researchers say that RESCUE potentially could be used as proactive wound healing treatment of acute injuries.    

Researchers are now actively developing new strategies to treat brain diseases given that CRISPR/Cas9 editing of neurons is difficult. RESCUE’s advancements are now helping tackle the gene variant APOE4 which has emerged as a consistent culprit of late stage development of Alzheimer’s disease. APOE4 differs from APOE2 by only two instanecs, most notably, APOE2 is not a risk factor for Alzheimer’s. Amazingly, when APOE4 RNA is imported into cells RESCUE can convert risk associated APOE4 to a non risk varient APOE2 sequence. 

As can be expected, researchers are eager to understand the full benefits of RESCUE as a clinical tool in treating disease mutations. Similar to recent CRISPR solutions developed, RESCUE system technology is slated to be broadly shared over the coming weeks, freely made available to nonprofit and academic research.

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