Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.

Billions of cells that were genetically modified with the powerful gene-editing technique called CRISPR have started working, as doctors had hoped, inside the body of the first sickle cell patient to receive the experimental treatment, according to highly anticipated data released Tuesday.

The edited cells are producing a crucial protein at levels that have already exceeded what doctors thought would be needed to alleviate the excruciating, life-threatening complications of the genetic blood disorder, the early data show. Moreover, the cells appear to have already started to spare the patient from the agonizing attacks of pain that are the hallmark of the disorder.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating the patient. "This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal

It's a miracle," says Gray, who says she has hope for the first time after a lifetime of struggling with excruciating pain and debilitating, life-threatening complications of the disease. Sickle cell disease is an inherited condition that is marked by defective oxygen-carrying red blood cells.
"When you pray for something for so long, all you can have is hope," says Gray, 34, who has four children. "It's amazing."

Fetal hemoglobin is a protein that is normally produced only by fetuses and newborn babies for a short time after birth. So scientists used CRISPR to edit a gene in bone marrow cells that had been removed from Gray's body.

The edited cells were infused back into her system, and the editing change allowed the cells to start producing fetal hemoglobin again. The hope is that the fetal hemoglobin will compensate for the genetic defect that has resulted in sickle cell disease and its abnormal form of adult hemoglobin.

"I think it's enormously exciting that we've reached a point where gene editing using CRISPR is being applied to sickle cell disease," says Dr. Francis Collins, director of the National Institutes of Health. Collins, who is not involved in the research, noted that sickle cell disease affects about 100,000 people in the United States and millions more worldwide.

NPR

wow!!!

This is a good news

liberalchick:
Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.

Billions of cells that were genetically modified with the powerful gene-editing technique called CRISPR have started working, as doctors had hoped, inside the body of the first sickle cell patient to receive the experimental treatment, according to highly anticipated data released Tuesday.

The edited cells are producing a crucial protein at levels that have already exceeded what doctors thought would be needed to alleviate the excruciating, life-threatening complications of the genetic blood disorder, the early data show. Moreover, the cells appear to have already started to spare the patient from the agonizing attacks of pain that are the hallmark of the disorder.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating the patient. "This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal

It's a miracle," says Gray, who says she has hope for the first time after a lifetime of struggling with excruciating pain and debilitating, life-threatening complications of the disease. Sickle cell disease is an inherited condition that is marked by defective oxygen-carrying red blood cells.
"When you pray for something for so long, all you can have is hope," says Gray, 34, who has four children. "It's amazing."

Fetal hemoglobin is a protein that is normally produced only by fetuses and newborn babies for a short time after birth. So scientists used CRISPR to edit a gene in bone marrow cells that had been removed from Gray's body.

The edited cells were infused back into her system, and the editing change allowed the cells to start producing fetal hemoglobin again. The hope is that the fetal hemoglobin will compensate for the genetic defect that has resulted in sickle cell disease and its abnormal form of adult hemoglobin.

"I think it's enormously exciting that we've reached a point where gene editing using CRISPR is being applied to sickle cell disease," says Dr. Francis Collins, director of the National Institutes of Health. Collins, who is not involved in the research, noted that sickle cell disease affects about 100,000 people in the United States and millions more worldwide.

NPR

^^^Brilliant... a giant leap for mankind.

RuggedSniper:
^^^Brilliant... a giant leap for mankind.

Indeed.