Artificial MIRNAS are potential gene therapy tools, especially for incurable monogenic disorders

Abstract

Well-designed artificial miRNAs (amiRNAs) are as effective as short hairpin RNAs (shRNAs) but produce 10–80 times less siRNA. They enable long-term silencing and are safer than other RNAi triggers. They are suitable instruments for gene therapy techniques, especially for incurable monogenic diseases. In clinical studies, stereotactic injection of AAV5 directly into the striatum is the most effective approach. Intravenous injections would not only make patients more comfortable, but would also reduce the cost of complex brain surgery.In terms of structure, biogenesis, and expression levels, Ami RNAs are more "natural" than other gene therapy methods. They also utilise the cell's native protein machinery and do not produce irreversible alterations, unlike genome editing technologies.  The amount of time spent on a technology determines its level of progression. ASOs have an edge in this regard, as seen by the number of authorized medicines. Perhaps RNAi is just around the corner