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Root cause of disease -

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?

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Genes at the center -

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.

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New era -

Emerging therapies, such as gene editing and replacement, pave the way for a new era in science and medicine.

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Experimental technologies -

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?

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RNA interference -

If you guessed RNA interference, you are right. The discovery of RNA interference (also known as RNAi therapies) earned scientists the Nobel Prize.

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Emerged in 2018 -

The first ever RNAi therapies emerged in 2018, developed by US-based pharmaceutical company, Alnylam Pharmaceuticals.

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Approved therapies by the FDA -

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.

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Changing the course of 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.

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Safety tested -

The therapies are being used by tens of thousands of people already, demonstrating consistent safety for human use and clear efficacy.

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Natural process -

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. 

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Instruction manual -

Our genes hold what is essentially an instruction manual to produce proteins. The proteins are responsible for “almost all cellular and body functions.”

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Reduce protein production -

Where does the RNAi come in? Well, the cells use the RNAi on genes to reduce the production of certain proteins.

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Mimic natural process -

Scientists are simply attempting to imitate this process. Genes that can either directly cause or  contribute to disease are therefore targeted with RNAi therapies.

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Disruption of production process -

RNAI therapeutics work by disrupting the production process of proteins involved in a particular disease.

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Nature does the work for you -

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.

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Going after root causes -

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.

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Root cause addressed -

By intervening in the production of proteins, the root cause that is perpetuating a disease or illness is directly addressed.

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Leaky tap -

In an article by Scientific American, the analogy fixing a leaky tap is used to better understand RNAi.

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Conventional medicines akin to mopping -

Through this analogy, we can understand that conventional medicines are like mopping the floor after a leak. 

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RNAi akin to fixing the leak -

In contrast, we can think of RNAi therapeutics as the intervention that actually fixes the leak, instead of just mopping up the aftermath.

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Silence genes -

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.

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Slow down disease -

For those facing diseases without a cure or viable treatment options, RNAi can slow down the progression of disease.

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Diminish and reverse disease progression -

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.

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What about gene editing? -

So what’s the difference between RNAi and other genetic interventions? Other innovations, such as CRISPR-based gene editing, change genes within the cells.

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Target RNA molecules -

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.

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No permanent DNA changes -

This makes RNAi therapeutics more attractive, in general, to modern science, as there are no permanent changes to DNA structure that accompany this intervention.

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Extensive testing -

This fact makes RNAi therapeutics safer and easier to normalize on the medicine market, as they have been through extensive testing and use.

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Successful in various treatments -

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.

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Source of hope -

For patients and caregivers who are facing limited treatment options or low survival rates, RNAi offers a great deal of hope.

Sources: (Scientific American)

See also: FDA approved: New medications and vaccines

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