![]() Watts, PhD, professor of RNA therapeutics and co-author on the study. “If you think of the cell as being a huge block of text in a word processing program, siRNA is like a search and find function using the right combination of letters you can find any word in the text, or in this analogy, any genetic sequence,” said Jonathan K. This prevents the cell from making proteins from that specific genetic sequence. Once bound to its target, the siRNA recruits cellular proteins that cut the mRNA, which is then degraded naturally by the cell before it can produce the corresponding protein. siRNA molecules interfere with the expression of genes by binding to messenger RNA (mRNA) after it’s been transcribed from the genome. They are part of the RNA interference (RNAi) system first identified by 2006 Nobel Laureate Craig Mello at UMass Chan Medical School. Small interfering RNA (siRNA) is a class of short, noncoding RNA molecules, only 20 to 24 base pairs in length, found in cells and that can be produced synthetically. “The lung is a tough organ to get RNA molecules to because it’s very sensitive to potential toxins and particles that can cause immune reactions,” said Hariharan. Hariharan and colleagues successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. This would potentially open a new class of therapeutics for treating lung diseases.” “We’ve shown sufficient silencing to prove anti-viral efficacy can be done with siRNA and we think this architecture is the way forward to using RNA silencing therapeutically in the lungs. Hariharan, PhD, co-author of the study and a postdoctoral associate in the lab of Anastasia Khvorova, PhD, the Remondi Family Chair in Biomedical Research and professor of RNA therapeutics. “Achieving robust silencing at this level that is well tolerated hasn’t been achieved before,” said Vignesh N. More importantly, the platform technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses. It is the first demonstration that multimeric siRNA molecules can be taken up into the lung after intranasal administration and achieve safe and robust genetic silencing. Published in The Proceedings of the National Academy of Sciences, the study by a multidisciplinary team of RNA biologists, chemical biologists, immunologists and virologists describes the delivery of siRNA molecules locally to lung tissue. Scientists at UMass Chan Medical School have developed a technology to deliver gene therapy directly to lung tissue through intranasal administration, a development that could potentially create a new class of treatments for lung disease.
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