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RNA - a novelty to Therapeutics

The scientists at Zurich have come up with an unique way to cure some rare hereditary diseases, caused by gene mutation. They are using RNA, being one of the central molecules for storing and carrying genetic information, to act on the specific organ’s RNA of the body to combat the effect of the gene mutation and bring it back to normal functionality.

The disease that is of main focus is erythropoietic protoporphyria (EPP) where the main enzyme ferrochelatase which produces haemoglobin (the oxygen supplier of blood) is produced in lesser quantities, leading to the accumulation of protoporphyrin. Protoporphyrin when reacts with sunlight produces molecules, attacking tissues and causing inflammation, when a person is exposed to light.

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The short RNA molecules produced in the lab bind to the RNA copy ferrochelatase gene, and they have seen the restoration of the production of these enzymes by the RNA molecules that nullify the effects of the mutation.

The main challenge they have faced is the RNA molecule to go and specifically bind to the organs RNA molecule of interest. For this specific disease EPP, they fused this RNA molecule with a molecule tested on EPP mice model, “Cholesterol” and the fused molecules could reach the bone marrow and do the job. This cannot be termed as an RNA drug yet as it's the preliminary stage of research.

The study at its first stage is very promising and the future goals of this study as mentioned by Jonathan Hall, Professor of Pharmaceutical Chemistry at ETH Zurich is to optimize the fusion molecule or examine other fusion molecules that can work effectively through more refined EPP Mouse models and then further the perfect drug candidate can be tested on humans. Together with Daniel Schümperli, Emeritus Professor from the University of Berne, and colleagues from ETH, University Hospital Zurich and Triemli Hospital Zurich, they have succeeded by coming up with the RNA molecule that can make up to the gene mutations in the bone marrow cells.

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