The 30 Year Wait for a Nobel Prize: MicroRNA

 

In 1993, two scientists, Victor Ambrose (professor of natural science at UMass Chan) and Gary Ruvkun (genetics professor at Harvard), made a discover that completely rewrites our understanding of gene regulation and genetics as a whole. Unfortunately, this discovery was met with "deafening silence from the scientific community" (Nobel Committee). Finally this year, the two were granted with a long-overdue Nobel Prize of Physiology or Medicine for their discovery of microRNA, and its role in post-transcriptional gene regulation. 

To further explain, the two began researching C. elegans (1-millimeter-long roundworm) in the late 1980s as postdoctoral fellows at MIT. After separating, they both continued to research the roundworm and Ambros made an interesting discovery. While looking into a mutation, he noticed a small fragment of RNA (microRNA) blocking the lin-14 gene by binding to mRNA. Before this discovery, it was believed that gene regulation was controlled only by transcription factors, which would give cells specialized purposes. Thus, skin cells would function as skin cells and not brain cells, as an example. Even facing criticism that this was unique to simpler organisms like the C. elegans, the two published this finding in 1993 and furthered their research into the animal kingdom. 

In 2000, the two found that the same mechanism of microRNA plays a titular role in gene regulation in many species in animals as well. Since then, they have identified over 1,000 different genes for microRNA in humans. Additionally, each microRNA regulates many different types of genes, as well as genes being regulated by many types of microRNA.  

These findings are nothing short of incredible and it is amazing that Ambrose and Ruvkun are getting the recognition for this discovery in such a big way. I am excited to see what will come next for the two, and it is always very intriguing to see the "laws" of genetics being rewritten even today. 

https://www.usnews.com/news/health-news/articles/2024-10-07/u-s-scientists-win-nobel-prize-in-medicine-for-discovery-of-mrna

MicroRNAs discovered as direct relation to intelligence in octopus brain

Octopuses also referred to as soft bodied cephalopods are invertebrates with a very complex nervous system. In a recent study referred to in the article; MicroRNAs are deeply linked to the emergence of the complex octopus brain , elevated RNA editing in the nervous tissue was related to RNA regulation and the large role it plays in cognitive function and success among this species. More specifically the miRNA were expressed more frequently in the adult's neuronal tissues and during development suggesting that the miRNAs are in direct correlation with the evolution of complexity in animal's brains. 

In agreeance that microRNAs are a key contributor to complexity in brains is Michael Le Page in the article; Octopus brains may have become complex the same way human brains did. High intelligence in many species is being directly correlated with high numbers of microRNAs and their ability to generate different types of neurons. Although, these invertebrates evolved completely separately from invertebrates the microRNAs both became present in animals with complex brains. I find it important to recognize others species intelligence and brain complexity in order to understand how humans and other species evolved into todays present species. It is also important moving forward how it will increase complexity and in what species. 

 

The Progression of Gene Therapy Intervention for Sickle Cell Disease

  

    Sickle cell disease affects thousands of people throughout the globe and is recognized to be prevalent in African populations. Early clinical trials centered around gene therapy for sickle cell disease have been researched by geneticists since the 1990s.The intent of gene therapy for sickle cell disease is to correct genetically modified dysfunctional hemoglobin that leads to the development of structurally altered red blood cells that block blood vessels and lead to complications in organ function. The ultimate goal of research trials centered around gene therapy intervention for sickle cell disease aimed to stimulate the body’s ability to produce fetal hemoglobin by insertion of microRNA. Through insertion of the microRNA into the body, the dysfunctional protein known to alter red blood cell structure was corrected and fetal hemoglobin was successfully produced.

From the research and clinical trials around microRNAs potential to alleviate symptoms associated with sickle cell disease, six participants of the study were shown to have successful outcomes. The research results provide a possible cure to sickle cell disease and hope to many who inherited the illness. 

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