In a First, US Patient Treated with Gene-Editing Tool CRISPR

It sounds like something out of a science fiction novel, but for the first time in the US, a patient with a genetic disorder has been treated with a tool that alters your DNA.

The incredible gene-editing technology – CRISPR – was used for the first time inside the body of a patient for this historic treatment.

34-year-old Victoria Gray was treated for her sickle cell anemia with CRISPR by doctors in the US, reported NPR.

"“It is just amazing how far things have come.”" - Victoria Gray

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Not So Fast, Say Doctors

Gray was the first patient to be publicly involved with CRISPR research, and Dr Haydar Frangoul, medical director at the institute she was treated in, said of the process, “This is a very big deal, this could benefit many patients.”

However, while the success of this type of treatment gives many patients great hope, doctors caution that recommending CRISPR as regular, safe treatment for all may take a little longer.

Gray and her medical progress would need to be monitored carefully for many years to determine the safety and efficacy of CRISPR first.

How Does CRISPR Work?

“It’s a good time to get healed,” Gray told NPR about living with a debilitating genetic disorder in a time of life-changing, revolutionary medical advances.

CRISPR enables doctors to edit and customise the human genome and presents a hopeful way forward to treat previously incurable diseases.

Gray was suffering from sickle cell anemia, a disorder that affects millions of people around the globe and mostly African-Americans in the US. It is a genetic disorder that causes the bone marrow to create hard, sickle-shaped red blood cells that get stuck inside blood vessels and can’t carry much-needed oxygen normally around the body.

This results in a host of complicated and often life-threatening issues. So for the treatment, doctors genetically modified cells from the patient’s own bone marrow to make them create a protein that, typically is produced by fetuses and babies for a small amount of time after birth.

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This is done to compensate for the defective protein that causes sickle cell anemia. Hopefully, scientists say, this procedure will enable patients to live a healthy life.

Exciting But Proceed With Caution, Say Medical Experts

Researches and medical experts saw the development as an exciting medical advancement but did call for further research on CRISPR.

"“CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases. It seems to work really well in animals and culture dishes. It’s completely unknown how it works in actual human beings. So there are a lot of unknowns. It might make you sicker.”" - Laurie Zoloth, a bioethicist at the University of Chicago.

Since it is a new technology, there are always going to be unknowns and risks but Dr Frangoul asserts that further research is ongoing slowly and steadily.

For now, Gray acknowledges the risks but adds that this experimental study “gives her hope if nothing else” to finally start living a full life.

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