Personalized medicine is about the customization of healthcare. With this concept, medical decisions, practices, and drug products are tailored to the individual patient. Here medical laboratory testing would select the appropriate therapy for an individual person based on a given person’s individual genetic make-up and particular physical characteristics. According to a recent tweet from the Mayo Clinic, “#biotech is revolutionizing personalized medicine.”
In a breakthrough, researchers at The Scripps Research Institute in Florida have devised broad methods to design precision medicines against diseases caused by RNA. RNA stands for ribonucleic acid. It is an important molecule with long chains of nucleotides and vital for a health organism. When RNA acts erratically this can lead to diseases occurring.
To combat such genetic diseases medical researchers have attempted to develop drugs to combat the errant RNAs. Much of this research has been unsuccessful because of sufficient effectiveness and due to side effects. However, now a breakthrough has been achieved.
According to lead researcher Professor Matthew Disney, who spoke with Laboratory Roots: “With the precision of a surgeon’s scalpel, we have shown that small molecules can be designed to seek out and destroy only disease-causing RNAs.”
As well as this increased precision, the researchers have developed novel chemical approaches to use a disease-causing RNA to help make its own drug. This is by using that RNA as a catalyst for drug synthesis at the needed site.
Trials have taken place on myotonic dystrophy type 1 (progressive muscle wasting and weakness). This disease is caused by an error in the RNA genetic code. A designer molecule can selectively recognize larger, disease-associated repeats over shorter, normal ones and alter the coding. This was advanced through fluorescence imaging to spot the errant code.
The research has been published in the journal Nature Chemical Biology, in a paper titled “Sequence-based design of bioactive small molecules that target precursor microRNAs.”
