RNAi technique, how far is it from pediatrics?

Abstract

The new technology of ribonucleic acid interference (RNAi) or small/short interfering RNA (siRNA) can be used to reduce expression of genes in a sequence specific manner, and thereby can treat various diseases caused by expression or overexpression of genes. | WH AT IS RNA INTERFERENCE TECHNIQUE?The RNA interference (RNAi) derived from observation that small or short double stranded RNA (siRNA) molecules, 19-22 base pairs in length, can interfere with the process of gene expression by degrading the mRNA of a gene in a sequence-specific manner. 5-7 The consequence of the interference is apparently reducing the expression of a particular gene, which is also described as down-regulation of gene expression. If the gene expression is reduced by greater than 70%, the term "gene silencing" is applied. Therefore, RNAi has genesilencing or gene inhibitory effect, and such effect has enormous therapeutic potential. [5][6][7] The RNAi-based therapeutic approaches have the advantages of specificity, potency, and versatility. 5To silence a gene, an siRNA of specific sequence which is complementary to the sequence of a certain segment of the target mRNA should be designed and synthesized. In most cases, the synthesized siRNA (naked siRNA) is packaged/encapsulated physically (eg, in liposomes or lipid nanoparticles) or conjugated or complexed chemically in a certain way so that the siRNA can be delivered to the sites or cells where they can act on the target mRNA without being degraded by ribonucleases (RNases). The naked siRNA can be directly injected into the tissue topically if this is needed and appropriate. ------------------------------------------------------This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children

“…Many of these diseases, including single-gene disorders, cancers, and infectious diseases, affect infants and children. 18 Although RNAi therapies have previously demonstrated efficacy and safety in adults, they have not been evaluated in infants and young children. This ILLUMINATE-B phase 3 multicenter study evaluates the use of an siRNA in infants and young children <6 years old.…”