NPR reported yesterday that CRISPR gene-editing technology was used for the 1st time to treat a patient with the genetic disorder sickle cell.
What is CRISPR again?
Right, but playing with DNA seems scary
Yup. The field is filled with landmines all over especially with the rogue scientists who are trying to create designer babies using the tech. Applying CRISPR at embryo level like that is especially dangerous because whatever alterations made will forever be passed down to generations. Imagine a never before seen DNA mutation being introduced to the human gene pool – you don’t really wanna risk that.
Hope for people with genetic disorders and cancers
However, CRISPR could be a very useful tool if used individually – in other words, the modifications are specific to the patient and won’t get passed down. Doctors at UPenn have been using the tech to treat cancer patients and starting this fall, Editas Medicine and Allergan will test CRISPR tech with up to 18 people to see if it could cure inherited blindness.
What is Sickle Cell Disease?
It’s a genetic disorder that causes bone marrow to produce a defective protein that makes sickle-shaped and sticky blood cells instead of round ones. The deformed cells get stuck inside blood vessels and don’t move oxygen normally. It causes debilitating pain, life-threatening complications, organ damages, and makes the patients more vulnerable to infections.
About 100,000 people in the US suffer from sickle cell and many people with the disease don’t live past their 40s. Legendary rapper Prodigy battled with sickle cell his whole life before passing away from complications at the age of 42 in 2017.
Take a quick listen to his song titled “You can never feel my pain” below to get an idea of how devastating sickle cell is.
“I’m talkin’ bout permanent, physical suffering
You know nothin’ about that…” – RIP Prodigy
How does the CRISPR treatment work?
The scientists at Sarah Cannon Research Institute extracted cells from the patient’s own bone marrow, edited them with CRISPR tech so that the cells can produce fetal hemoglobin, which is produced by fetuses in the womb to provide oxygen but disappears after birth, and injected them back to the patient.
They’re trying to see if the fetal proteins made in the modified cells could compensate for the defective proteins that produce sickle-shaped blood cells and ultimately, if the treatment could be a valid option for patients to replace bone marrow transplant, which carries the risk of transplanted cells crashing within patient’s body.
This is just a start and it will probably take years of close monitoring to see if the tech really works but we can’t help but wish Prodigy was here to see this development.