/The future of CRISPR is now

The future of CRISPR is now

Summary: CRISPR is revolutionizing experimental therapies for genetic disorders ranging from sickle cell disease to blindness, but where should society draw the line in editing genes?

Original author and publication date: Bridget Balch – December 2, 2021

Futurizonte Editor’s Note: The future is now. Period. And nobody is inviting us to be part of the new future.

From the article:

It has been more than a year since Markus Mapara, MD, a professor of medicine and director of blood and marrow transplantation at Columbia University Irving Medical Center in New York, first used an experimental CRISPR gene-editing treatment in a patient with sickle cell disease, an inherited blood disorder that can cause severe pain, organ damage, and premature death.

Although the clinical trial is still in early stages and has only been tested in a few patients, so far, the results are promising.

“[The patient is] doing phenomenally well,” says Mapara, who is a hematologist, oncologist, and blood transplant physician. “He has not had a single crisis.”

Sickle cell disease, which currently affects about 100,000 people living in the United States and millions worldwide, is the result of a genetic mutation that produces an abnormal type of hemoglobin, the protein that red blood cells use to deliver oxygen throughout the body. The abnormal cells take on a sickle, or curved, shape, which can clot within narrow blood vessels.

“This is a huge health problem for these patients,” Mapara says. “It has a huge impact on their quality of life and well-being.”

At the moment, a bone marrow transplant from a healthy donor is the only curative option, but this approach can have severe complications.

“You’re running into risks of introducing foreign cells into a recipient,” Mapara says. “[The body] may reject them, or the donor cells might attack the recipient.”

But with the development of CRISPR (which stands for clustered regularly interspaced short palindromic repeats), new opportunities for treatment using the patients’ own cells have opened up.

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