The possibilities for intervention are endless and alongside the use of mRNA as vaccines there is also the use of mRNA as a template to produce missing or deficient proteins. High hopes for the treatment of rare genetic diseases
The drugs of the future will be more and more based on RNA: by choosing the right one, the cells can be produced missing proteins, for example in the case of genetic diseases, or otherwise blocking the proteins that lead to some trouble. A revolutionary approach because, instead of taking a drug that is still a foreign molecule, the biology of the organism is exploited to create what we need or to change some cellular functions to our advantage.
Even in heart disease
The possibilities of intervention are endless and alongside the use of mRNA as vaccines there is also the use of mRNA as a template to produce missing or deficient proteins: for example, we are studying whether a synthetic mRNA can increase the quantity of a vascular growth factor and thus be of help in patients with heart failure undergoing coronary artery bypass grafting; or whether an mRNA drug can help in a rare disease, methylmalonic acidemia, by instructing cells to produce the defective enzyme responsible for this rare disease.
Abnormal proteins
One of the most interesting applications, for which there are already approved drugs, for the technology of RNA interference, or RNA interference: the result of research that led to the Nobel Prize for Medicine in 2006, is to create small synthetic RNAs that they fit precisely on specific cellular mRNAs silencing them, that is, preventing them from being translated into proteins. The result is the shutdown of genes that if expressed would lead to harmful consequences, effectively solving many problems at the root because, instead of addressing the problems due to abnormal proteins, they are prevented from being produced.
Medicines
what happens with two drugs already approved for two rare diseases, patisiran for hereditary transthyretin amyloidosis, a disease in which an altered transthyretin accumulates and progressively damages organs, and givosiran for acute hepatic porphyria, in which the interfering RNA serves to block a liver enzyme that in hyperactive patients and produces a excess of toxic substances. While lumasiran is coming for primary type 1 hyperoxaluria (also in this case the expression of an enzyme that produces too much oxalate is blocked with deleterious effects), to treat hypercholesterolemia that does not respond well to other therapies already available in some Countries, and soon also in Italy, inclisiran: through a gene interference mechanism it inhibits the production of a protein that decreases the absorption of LDL cholesterol from the circulation, favoring this absorption thanks to a greater number of cholesterol receptors expressed on cells .
Targets
In short, the road appears to have been traced and the possible targets are so many that according to a study published in Nucleic Acid Research artificial intelligence is needed to identify the most suitable ones: faced with an enormous amount of mRna transcribed from the genome (85 percent of all transcribed DNA, even if much less are the genes translated), to find the right ones to modulate a titanic enterprise in which supercomputers will be increasingly indispensable.
April 11, 2022 (change April 11, 2022 | 19:20)
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