Mother of four is the first in the US for a controversial treatment of gene-processing sickle cells

A 34-year-old mother of four is the first American to volunteer to have their genes processed with the controversial CRISPR technology to treat her sickle cell disease.

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Victoria Gray van Forest, Mississippi, has suffered from her debilitating blood disorder all her life and often has so much pain that she cannot lift a spoon, she said NPR.

Sickle cell disease affects as many as 100,000 Americans – a disproportionately large number of whom are black, such as Gray – and while treatments can relieve symptoms, they do nothing to tackle the underlying disease.

The experimental gene processing technology, known as CRISPR, can change that by altering Gray & # 39; s DNA to produce a kind of protein patch for her condition, but it is impossible to say whether there is additional damage to her genes is.

Scientist Sat Sarah Cannon Research Institute will use the controversial CRISPR tool to perform a sort of & # 39; cut-and-paste & # 39; procedure on Victoria Gray's 34-year-old DNA, hoping for a fix for her debilitating sickle cell disease in her programmable genes

Scientist Sat Sarah Cannon Research Institute will use the controversial CRISPR tool to perform a sort of & # 39; cut-and-paste & # 39; procedure on Victoria Gray's 34-year-old DNA, hoping for a fix for her debilitating sickle cell disease in her programmable genes

With four young children, all under the age of 13, sickle cell disease makes basic mother's tasks sometimes unbearable for Gray.

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& # 39; It's terrible, & # 39; said Gray, a mother who stayed at home, to NPR.

& # 39; If you can't walk or lift a spoon to feed yourself, it becomes very difficult … & # 39; Sometimes it feels like a lightning strike in my chest. & # 39;

Gray has a genetic mutation that deforms the red blood cells that transport oxygen through her body.

HOW DOES CRISPR DNA PROCESSING WORK?

The CRISPR gene processing technique is increasingly used in health research because it can change the building blocks of the body.

At a basic level, CRISPR works as a process for cutting and pasting DNA.

Called technically CRISPR-Cas9, the process involves sending new DNA strands and enzymes to organisms to process their genes.

In humans, genes act as blueprints for many processes and traits in the body – they dictate everything from the color of your eyes and hair to whether or not you have cancer.

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The components of CRISPR-Cas9 – the DNA sequence and the enzymes needed to implant it – are often sent into the body via a harmless virus, so scientists can determine where they are going.

Cas9 enzymes can then cut DNA strands, effectively disable a gene, or remove portions of DNA that need to be replaced by the CRISPR's. These are new parts that are submitted to change the gene and the effect of which is preprogrammed to produce.

But the process is controversial because it can be used to change babies & # 39; s in the womb – initially to treat diseases – but can lead to an increase in & # 39; designer baby & # 39; s & # 39; because doctors offer ways to change the DNA of embryos.

Source: Broad institute

Instead of round discs with smooth edges, many of the cells that her bone marrow produces are sickle-shaped or crescent-shaped red blood cells.

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This form is & # 39; sticky & # 39 ;, in microbiological terms, meaning that they do not easily pass through smaller blood vessels.

As a result, much of Gray & # 39; s body is starving for vital oxygen, and the sickle cells periodically build up in her blood vessels, causing her huge pain and damaging her organs.

The genetic trait that causes sickle cell disease is much more common in black and African-American people, one in 13 of which is born with it, estimates the Centers for Disease Control and Prevention.

And one in 365 African-American and black babies are born with the disease itself and many will die in their 40s.

This defective DNA code means that Gray & # 39; s bone marrow has and will continue to produce unhealthy red blood cells throughout its life, with no hope of relief, except by painkillers and blood transfusions (bone marrow transplants offer better treatment, but are usually done only in children with severe symptoms).

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But she may be the first American for whom that is not true, if the experimental treatment she is going to undergo works.

Gray volunteered for a test conducted by the Sarah Cannon Research Center in Nashville and Vertex Pharmaceuticals.

The goal is to use CRISPR to process its DNA and to convert a genetic switch that usually only & # 39; to & # 39; is for newborns to make new hemoglobin, the oxygen-carrying protein.

If it works, the fetal protein can be a lifelong solution for Gray and hundreds of thousands of sickle cell patients around the world.

The treatment is not without risk, but bioethicist Dr. Arthur Caplan of the University of New York sees it with cautious optimism.

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& # 39; I tend to say: & # 39; Yes & # 39 ;, you should try this, & # 39; said Dr. Caplan against DailyMail.com.

& # 39; It is a good disease to set up, it is a miserable, painful disease that really impedes quality of life and affects thousands of Americans and (more) globally, so it can really be a blessing & # 39; for the treatment of a large population.

Gray & # 39; s condition means that many of its red blood cells are in the form of sticky sickles (center), rather than smooth, round and oxygen-rich discs

Gray & # 39; s condition means that many of its red blood cells are in the form of sticky sickles (center), rather than smooth, round and oxygen-rich discs

Gray & # 39; s condition means that many of its red blood cells are in the form of sticky sickles (center), rather than smooth, round and oxygen-rich discs

CRISPR has been used to treat cancer patients in the US, but never to treat a completely hereditary disease such as sickle cell.

In France, scientists reported that a gene therapy after 15 months in 2017 did not show any & # 39; signs of disease & # 39; had left, but CRISPR, the most advanced and controversial genetic treatment to date, was not used in that case.

The technology, which performs a hyper-precise cutting and pasting maneuver on DNA, came under fire when Chinese scientists edited the genomes of a few twin babies and triggered an international wave of caution to technology.

& # 39; But remember that this (sickle cell treatment) is not a germ line, as the Chinese scientists did, it is somatic (not hereditary), so the risk of passing it on to other people if there is some kind of unwanted changes is incorrect , & # 39; says Dr. Caplan.

& # 39; That said, it's risky for the subject. & # 39;

Although animal and petri dish tests have been successful enough to give the Food and Drug Administration the go-ahead for live human testing, it has never been done before and it is possible that CRISPR may adversely affect unwanted DNA changes.

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Dr. Caplan is concerned that the private company running the trial, Vertex, could postpone trials to amaze its investors, and is concerned that, if it works, treatment will be prohibitive for those who need it the most.

Nevertheless, he says sickle cell disease is a valuable condition to try out CRISPR because it can have a major impact on public health.

In his opinion Gray seems to be a well-informed and willing candidate to take the plunge – and she seems to feel the same.

& # 39; It's a good time to be healed, & # 39; she said to NPR.

. (TagsToTranslate) Dailymail (t) health

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