This infection is one of the most common causes of death worldwide
Medical research is on the edge of innovation, focusing on what is called genetic medicine. Scientists are placing greater emphasis on both the genetic blueprint and therapies that target genes as the most effective methods to target the root cause of disease.
Medical technologies are quickly adapting and offer diverse methods to go about this strategy, but one of the key therapeutic modalities that scientists are placing their bets on is RNA interference (RNAi) therapeutics.
Want to learn more about this innovative medicine? Click through to find out more.
What if we were to tell you that the root cause of all disease is your genes? Does the complexity of your genetic blueprint make you weary or relieved?
In many ways, it might come as a relief. Researchers are wrapping their heads around the science that puts genes at the center of therapeutic interventions.
Emerging therapies, such as gene editing and replacement, pave the way for a new era in science and medicine.
While there are a great deal of experimental medical technologies now targeting genes, there is one that is quite established. Do you know what it is?
If you guessed RNA interference, you are right. The discovery of RNA interference (also known as RNAi therapies) earned scientists the Nobel Prize.
The first ever RNAi therapies emerged in 2018, developed by US-based pharmaceutical company, Alnylam Pharmaceuticals.
Since 2018, five more RNAi therapeutics have been approved by the US Food and Drug Administration (FDA), marking a clear shift in how scientists are looking at disease.
Scientists working in the field are confident in the potential that RNAi holds to change the course of disease progression for millions of patients.
The therapies are being used by tens of thousands of people already, demonstrating consistent safety for human use and clear efficacy.
So what are RNAi therapies and how do they work? RNAi is a natural process. This process takes place in each of the body’s cells.
Our genes hold what is essentially an instruction manual to produce proteins. The proteins are responsible for “almost all cellular and body functions.”
Where does the RNAi come in? Well, the cells use the RNAi on genes to reduce the production of certain proteins.
Scientists are simply attempting to imitate this process. Genes that can either directly cause or contribute to disease are therefore targeted with RNAi therapies.
RNAI therapeutics work by disrupting the production process of proteins involved in a particular disease.
In this way, there is a natural flow to the progression of medicinal intervention, as the imitation of a natural process allows “nature [to do] the work for you,” according to a biochemist at UT Southwestern Medical Center.
This is what makes RNAi not only innovative, but also foundational. Instead of merely targeting symptoms, the RNAi modality goes after root causes. Pictured is an RNAi treatment that targets macular degeneration.
By intervening in the production of proteins, the root cause that is perpetuating a disease or illness is directly addressed.
In an article by Scientific American, the analogy fixing a leaky tap is used to better understand RNAi.
Through this analogy, we can understand that conventional medicines are like mopping the floor after a leak.
In contrast, we can think of RNAi therapeutics as the intervention that actually fixes the leak, instead of just mopping up the aftermath.
Advocates of genetic medicine believe RNAi is poised to “silence virtually any gene.” For patients of all kinds, this offers a meaningful sense of hope.
For those facing diseases without a cure or viable treatment options, RNAi can slow down the progression of disease.
Studies show that not only can RNAi interventions diminish a disease’s violent spread, but it can reportedly also stop progression altogether and even reverse its progression.
So what’s the difference between RNAi and other genetic interventions? Other innovations, such as CRISPR-based gene editing, change genes within the cells.
RNAi therapeutics do not actually make changes to the genes. They instead merely target and lower the efficacy of RNA molecules in the production of particular proteins.
This makes RNAi therapeutics more attractive, in general, to modern science, as there are no permanent changes to DNA structure that accompany this intervention.
This fact makes RNAi therapeutics safer and easier to normalize on the medicine market, as they have been through extensive testing and use.
RNAi therapeutics have been successful in the treatment of rare genetic diseases as well as common conditions, such as the driving factors behind cardiovascular diseases like high blood pressure.
For patients and caregivers who are facing limited treatment options or low survival rates, RNAi offers a great deal of hope.
Sources: (Scientific American)
Innovative new gene therapies target the root causes of disease
RNAi therapies are entering the market with a clear vision
HEALTH Rnai
Medical research is on the edge of innovation, focusing on what is called genetic medicine. Scientists are placing greater emphasis on both the genetic blueprint and therapies that target genes as the most effective methods to target the root cause of disease.
Medical technologies are quickly adapting and offer diverse methods to go about this strategy, but one of the key therapeutic modalities that scientists are placing their bets on is RNA interference (RNAi) therapeutics.
Want to learn more about this innovative medicine? Click through to find out more.