A breakthrough in technology could pave the way for the next generation of RNA-based therapies. DART VADAR is a responsive RNA sensor developed by researchers at Harvard University and MIT that uses an enzyme that edits RNA in the human body to activate the translation of a genetic payload in response to the presence of a specific molecular marker of disease and/or cell type.

According to the researchers, this technology has the potential to broaden the range of conditions that can be addressed with RNA-based therapeutics and enable the development of highly specific treatments for a variety of diseases. DART VADAR is based on the enzyme adenosine deaminases acting on RNA (ADAR), which binds to double-stranded RNA molecules and performs a specific base edit, converting a mismatched adenosine molecule to inosine and thus destabilizing the dsRNA structure.

The researchers created a single-stranded RNA circuit with multiple modular elements, including a sequence coding for a fluorescent green protein as an observable signal molecule that could be used instead of the UAG sequence. The researchers confirmed that the DART VADAR sensor worked and that its activity was low, so they added the ADAR gene sequence to their sensors to ensure that the sensor could work in different cell types.

This technology is a clinically relevant, compact RNA-based circuit that allows one to direct therapies to specific cell types and cells in specific states, minimizing off-target effects.

Source: Nature (link)
Images: MidJourney, Prompts by Lewis Farrell

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