Zach Winn's recent article in MIT News discusses how CAMP4 Therapeutics targets regulatory RNA. Many diseases are caused by dysfunctional gene expression, which produces too much or too little protein. The CAMP4 approach is to edit genes by inserting new genetic snippets into cells.
MIT professor Young and the CAMP4 co-founder have found that the regulatory RNA controls how much of the gene is expressed when molecules interact with transcription factors. This treatment could help metabolic diseases, heart conditions, and neurological disorders. They found that targeting RNA regulators could offer more precise treatments to target these conditions. The first CAMP lead drug candidate targets genetic defects in the metabolism of excreting ammonia. As the company plans to expand the study for preclinical safety, they hope to next look into seizure disorders. This can lead to learning about multiple conditions and opening up collaboration to make an important impact.
I looked on the CAMP4 website for further details on how the treatment is performed and couldn't find an answer to my question. I'd like to know if, despite the RNA regulators being precise in targeting the right genetic defects, the treatment could affect other genes and possibly lead to mutations.
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