RNA Therapies
RNA (Ribonucleic acid) could be designed to target specific genetic
diseases and disorders. This would treat the disease itself and not just the
symptoms. RNA therapies are new and perhaps unconventional, but there is no
doubt about the potential for RNA-based therapies in treating diseases in the
future.
RNA Structure
It is easy to be carried away by the potential and it must
be noted that RNA is an unstable molecule. RNA is a strand of phosphates;
ribose and nucleotides attached side-by-side by strong phosphodiester bonds
(formed in condensation reactions). Many viruses possess RNA as their genetic
material. Viruses reach target cells and inject their RNA into cells; this
causes the local cells to malfunction or not work at all, triggering an immune
response.
RNA Interference
RNAi
(RNA interference) occurs in nature. This is how cells control their gene expression.
siRNA (Short interfering RNA) disrupts this process by activating an enzyme
that cuts mRNA (messenger mRNA) into short pieces that can not be read.
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Pathway for miRNAs,
shRNAs, and siRNAs
Delivery of RNA therapies
RNA is typically a small molecule and is difficult to
produce in large quantities. pRNA (packaging RNA) is produced when small
molecules of RNA are assembled together. Through nucleic acid origami
techniques, small particles could deliver RNA directly to tumours, turning off
any cancer genes.
Further Reading:
RNA-Based Therapeutics: Current Progress
and Future Prospects : http://download.cell.com/chemistry-biology/pdf/PIIS1074552111004595.pdf?intermediate=true
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