CLEVELAND, Oh. - Danielle Lees adventurous spirt shines through, but behind the smiles of this aspiring actress is an unimaginable pain.

I went from like screaming, crying, please don't let me die to I just wanted the pain to be over. It is very sharp pain most of the time, to the point where holding my cell phone can hurt, Danielle painfully recalled.

Danielle was diagnosed before birth with sickle cell disease, where abnormal hemoglobin makes red blood cells rigid and shaped like sickles. These cells die early, leading to a chronic shortage of healthy red blood cells which are essential for carrying oxygen throughout the body.

The blood, basically, doesnt go to where it should be. So, the oxygen doesn't go to potential parts of the body, explained Dr. Rabi Hanna, pediatric oncologist at the Cleveland Clinic.

Sickle cell causes severe pain in the bones and can impact a patients heart, lungs, eyes and even cause strokes.

Dr. Hanna says medications can relieve some of the symptoms. Chemo also helps. Bone marrow transplants work when theyre not rejected.

But now, a one-time gene editing cell therapy is able to modify a patients own blood-forming stem cells to correct the mutation responsible for sickle cell disease.

We attach the patient to apheresis machine that's able to separate the stem cell from the red blood cells from the plasma, Dr. Hanna said.

Once the stem cells are collected and sent to the lab for gene editing, patients undergo chemo to destroy their remaining bone marrow, then the edited stem cells are infused back into their body.

The cells, they can go to their bone marrow and they start to build their house that will produce new white blood cells, new red blood cells, Dr. Hanna further explained.

Results have shown new white blood cells in patients at four weeks with no severe adverse effects.

The patients have also been free of sickle cell diseases pain attacks for an entire year. Danielle was one of the first in the CRISPR gene editing clinical trial.

I have so much more energy, thank God, Danielle said with relief.

The average life of a sickle cell patient is mid-forties. Doctors hope the CRISPR gene editing technology will change that and allow patients to live a long, pain-free life.

The Ruby clinical trial aims to enroll 40 more patients ages 18 to 50 with severe sickle cell disease.

More information is available at clinicaltrials.gov

View original post here:
Health Beat: A possible solution for sickle cell disease - 69News WFMZ-TV