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Exploring RNA DrugS: Antisense, RNAi, and MicroRNA

by | Jan 3, 2024 | Biotech for Non-Scientist

RNA-based drug therapy has taken the biotech world by storm. It has reshaped treatment landscapes through its ability to target “undruggable” areas. Furthermore, it offers personalized patient solutions. Therefore, this approach has the potential to revolutionize the future of medicine.

The Central Dogma

Gene expression is how DNA instructs cells to make proteins. Hence, proteins are essential for our body to grow and function, but proteins can also cause disease. 

RNA as the Middleman

DNA is so fragile that it remains in the nucleus, a protected space in the center of the cell. Inside the nucleus, the job of RNA is to transcribe the DNA into RNA. When RNA leaves the nucleus, it heads to the ribosome, where the ribosomes translate the RNA into proteins. Sometimes, that RNA codes for a disease-causing protein. RNA-based drugs prevent the RNA code from reaching the ribosome, stopping the production of disease-causing proteins.  

Antisense RNA Drugs Explained

Antisense RNA drugs are short, synthetic pieces of RNA whose sequence complements the RNA associated with disease-producing proteins. These enter patients’ cells and bind to the disease-causing RNA. This triggers the enzyme RNAse H to destroy the antisense RNA structure, stopping illness in its tracks!

RNAi Drugs Explained

RNAi is a complicated RNA drug. The body produces it after a series of steps. 

  • First, a disease-specific double-stranded RNA (dsRNA) is injected into a patient. 
  • Then dsRNA is processed into a “short interfering RNA” (siRNA) by the enzyme DICER. 
  • The siRNA binds to a second enzyme called RNA-Induced Silencing Complex (RISC), forming a siRNA+RISC complex. 
  • Lastly, siRNA+RISC complex attaches to and destroys the disease-associated RNA, nipping disease in the bud.
Recorded Biobasics 101

RNAi in Action

  • Genzyme’s Kynamro lowers LDL cholesterol levels by blocking the production of the protein apolipoprotein B.
  • Alnylum’s Amvuttra treats polyneuropathy of hereditary transthyretin-mediated amyloidosis by blocking the production of the protein transthyretin.

MicroRNA Drugs Explained

MicroRNA (miR) is a double-stranded RNA (dsRNA) that regulates protein expression. Like RNAi, DICER and RISC process miR into a single-stranded RNA. This newly formed RNA binds to the disease-associated RNA with its complementary sequence. Since miRs only bind to one end of the RNA, each miR can regulate multiple disease-associated RNAs, halting the disease. 

While no microRNA drugs have received FDA approval thus far, preclinical research on their potential applications for cancer is ongoing.

Biotech’s Darling

Traditional medicines require the drug to bind to the active sites of disease-causing proteins. However, only 10-14 percent of proteins can be targeted, leaving most diseases undruggable. Subsequently making RNA drugs the darlings of biotech because they prevent disease-causing proteins from ever being made, making the scope of treatment limitless.

Back to School

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