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Publication
RNA Therapeutics: How Far Have We Gone?
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Advisor(s)
Abstract(s)
In recent years, the RNA molecule became one of the most promising targets for therapeutic intervention. Currently, a large number of RNA-based therapeutics are being investigated both at the basic research level and in late-stage clinical trials. Some of them are even already approved for treatment. RNA-based approaches can act at pre-mRNA level (by splicing modulation/correction using antisense oligonucleotides or U1snRNA vectors), at mRNA level (inhibiting gene expression by siRNAs and antisense oligonucleotides) or at DNA level (by editing mutated sequences through the use of CRISPR/Cas). Other RNA approaches include the delivery of in vitro transcribed (IVT) mRNA or the use of oligonucleotides aptamers. Here we review these approaches and their translation into clinics trying to give a brief overview also on the difficulties to its application as well as the research that is being done to overcome them.
Description
Keywords
Humans RNA Splicing RNA, Messenger RNA, Small Interfering Genetic Therapy Oligonucleotides Oligonucleotides, Antisense Doenças Genéticas
Pedagogical Context
Citation
In: Romão L (ed). The mRNA Metabolism in Human Disease. Advances in Experimental Medicine and Biology. Springer, Cham., 2019, pp. 133-177. (AEMB, volume 1157). doi:10.1007/978-3-030-19966-1_7
Publisher
Springer Verlag