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4:37 PM
Living medicine: engineering bacteria for the treatment of genetic diseases

Pedro Belda ferré, California Institute, San Diego

(Speaking) a pill that has millions of microbes ready to colonize your gut has the potential to be a horror to a multitude. But it has the ability to freeze with effective fresh disease control equipment.

In many hereditary genetic diseases mutated gene means, in fact, that a person is not able to make relevant weighty substance, important for the rise, formation or functioning of their own body. At times, it is possible to straighten a synthetic substitute-a pill that they have every chance to take on every day, in order to change the fact that their corpse had to be arranged in a natural way. People with the rarest genetic disease, called phenylketonuria (PKU), there is no enzyme, important for protein breakdown. Without it, toxic chemical drugs accumulate in the blood and have all the chances to cause irreversible brain damage.

Luckily, it's easy to fix. Doctors cure the disease by putting their own patients on an ultra-low protein diet for the rest of their lives. Indeed, because the amendment was so common, PKU was the first disorder in which newborn children periodically inspected, since 1961, examining a drop of blood collected from the injection on the heel of the baby.

But suppose for yourself how difficult it is to measure everything that you eat in the direction of your entire life. To heal PKU, scientists are real-time exploring fresh healing strategies. Raz involves the introduction of gene editing tools to correct genetic mutations. However, today's development is still considered risky; there is a possibility of violation of other genes and causing concomitant harm to patients.

Actually what if it were possible to swap out a bad gene without affecting the genome of the patient? This is exactly what scientists from the Cambridge, Massachusetts biotechnology firm Synlogic created. They decided, in fact, that instead of this, in order to directly get involved in the human genome, they will include therapeutic genes in natural bacteria that are present in the human intestinal tract. These genetically modified bacteria then produce enzymes, which are not enough for patients with PKU, and break down proteins into non-toxic products.

I am considered a UCSD doctoral student who studies the Association of bacteria living in our body and how they affect our well-being. Now we are beginning to perceive the role they play in maintaining well-being. The next step is to find out how we can change them to make us feel better. And the study of Synlogic brings this dream one step closer.

Engineering bacteria living in our gut

You may be very surprised to ask that our intestinal tract is actually populated by trillions of microbes that can help us digest food, make vitamins for us, and nurture our immune system. This Association of microbes-our microbiome. Together they contain millions of different genes in their own genomes, outnumbered by our human genes 150 to 1, and we can apply them to our personal interests.

Escherichia coli Nissle 1917 is once of those bacteria living inside the greatest part of us and is extensively applied as a probiotic for over a century, confirming its own protection.

This microbe which Synlogic has chosen for the creation of fresh therapeutic "super bacteria" under the title SYNB1618 for patients with PKU.

Scientists have introduced the gene 3 which give the opportunity СЫНБ1618 to convert times from the build blocks of protein, amino acid called phenylalanine in the non-hazardous compound, phenylpyruvate. While the degree of phenylalanine remains low, patients with PKU show virtually no signs and live a normal life.

Non-hazardous if the GM-bacteria?

Enemies at the gene level of modified organisms have every chance to protest against the addition of designer bacteria in our guts. But as one such as they do with genetically modified foods, there will only be FDA regulations that ensure actually that these viruses are harmless.

In the case SYNB1618 researchers removed the gene that serious for the creation of an important component for the creation of microbes. If scientists do not provide the missing component for engineering microbes, they will not be able to reproduce and die. This is a method for surveyors to keep SYNB1618 under control in the patient's body.

When they tested the viruses on the mice, they noticed, in fact, that after 48 hours without the main component SYNB1618 disappeared from their intestines.

Scientists Synlogic even gained additional precautions when developing SYNB1618 and selection of bacteria for therapy. With the exception of genes added for such in order to cultivate phenylalanine, engineered bacteria contain literally the same genes as the first Nissan 1917 E. coli that is native to the gut, ensuring its own protection.

Does it really work?

As soon as the scientists proved that the bacteria have all chances to convert phenylalanine in the laboratory, they decided to install the bacteria in mice with the support of PKU. The results have shown that SYNB1618 actually aggravated phenylalanine circulating in the intestinal tract of animals, which reduced the value in the blood of treated mice.

After that, getting ready for tests on people, scientists tested SYNB1618 on chimps in order to ensure the security and efficiency of the people. Healthy monkeys without PKU were saturated with phenylalanine, and after that a dose of bacteria was isolated. Synb1618 bacteria have safely lowered the degree of phenylalanine in the blood-just as they have created in Mice.

Synlogic in real time checks SYNB1618 in men in phase 1 clinical testing.

This is a step towards a brand new therapeutic layout that invites tremendous potential for healing these human diseases like diabetes and cancer, as well as for predicting the value of inflammation in inflammatory bowel diseases.

As we discover and understand the role of all the bacteria that inhabit our bodies, I expect, in fact, that we will identify viruses that have every chance of being perfect transport methods for all sorts of gene therapies that cure even more diseases, covering the metabolism and the Central nervous system.

This article is published from the conversation under Creative Commons license. Read an unusual note here: http://theconversation.com/living-drugs-engineering-bacteria-to-treat-genetic-diseases-105796 ahhh!

Copyright 2018 Associated Press. All rights reserved. This fabric does not have the ability to be placed, broadcast, written or distributed.

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