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RNA “A path to cure disease”

by Maryam Ayyaz

RNA known as ribonucleic acid important molecule for flow of genetic information, used to treat many pathologies and for a diagnostic purpose such as for cancer biopsies. RNA a 3D structure consist of bases with complementary sequences and pairing can be done by a hydrogen bond RNA based molecules are used to guide biological pathways which termed as RNA-BASED Drugs.

The two most important factors for the production of RNA-BASED DRUGS, first one is nucleic acid chemistry and delivery method and the second one is the new target site or molecule. When RNA used to target a specific disease called as targeted drug because it targets the molecule of disease that cannot be treatable by other drug therapies.

Many hurdles came across for the production of RNA- based drugs, the following hurdles are 1) intracellular delivery across cell and endosomal membrane 2) poor pharmacokinetic properties 3) activation of innate immune nucleic acid sensors 4) off target effects such as suppression of unintended homologous targets, activation of RNA repair pathways and translocation or imprecise gene editing. So many recent advancements such as bioinformatics, separation sciences and nanotechnology can overcome the hurdles.

For coding of RNA splicing, multiple transcription and polyadenylation sites from genes are used. Two newly drugs have been approved by US Food and Drug administration in which two were Antisense oligonucleotides. RNA therapies divided in three groups that target nucleic acids in coding of proteins and the hybrid one as a RNA based mechanism into single packing.

Two main approaches are used for RNA drugs, first one is Antisense RNA where short oligonucleotides recognize and hybrid to complementary sequence in endogenous RNA transcript and alter their processing and the other one is mRNA encode to proteins for example mRNA for SARS (COVID-19).

Types of RNA therapy

Antisense oligonucleotides recognize the expression of target mRNAs by a specific binding for example fomivirsen for the treatment of cytomegalovirus. Two classes most commonly used for ASOS first one is H dependent ASOs which hydrolyze RNA and the other one is RNase H independent. ASOS delivery route is subcutaneous, intrathecal and intravenous.

Small interfering RNAs (siRNAs) which are small endogenous RNAs form a complex with protein to produce and expression of the target mRNA by a specific sequence binding .

Aptamers are nucleic acid which bind to specific proteins to provide their functions for example pegaptanib used to treat age associated macular degeneration which causes retina degeneration. RNA aptamers is most commonly used in surgery and emergency medicine because of their quick action as a targeted protein.

Messenger RNA in which exogenous mRNAs translated to functional proteins such as COVID-19 vaccine. Messenger RNA delivery route is intravenous.

Messenger RNA enhanced cell therapy enhance patient cell bind and encode the desire protein. It reinfused the patient cells and treat the specific disease.

Delivery mechanism of RNA

Lipid based liposome consist of polar head and non-polar tail. It consists of loading strategies and surface modulation. Most flexible drug delivery mechanism for various sites.

Polymer nanoparticle is a synthetic complex silica consist of silicon dioxide matrix mesoporous has high efficacy in drug delivery and suppression of tumour protein. Silicon nanoparticles is used in both genetic and chemotherapy agent as a combination drug.

Gold nanoparticles most commonly as a topical nucleic acid based RNA focused to delivery at a dermal site.


Advantages of RNA are as following, targeting non-drug able targets, fast production, long term effects for rare diseases, no risk of Geno toxicity, simple and quick design, no need of refrigeration for stability, synthesize chemically, used for target protein up to six months and low immunogenicity.


RNA based drugs in which RNA as a central pillar used to develop protein through the modulation binding effect. Act at a specific targeted site and used to produce desire effect and treat a disease. Now present it overcomes the convention drugs due to its vast rapid action and high efficacy.


Maryam Ayyaz, MS Industrial Biotechnology


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