The aim of the clinical trial was to find a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options.
18 patients, two of whom were treated at Cleveland Clinic Children's in the US, underwent a procedure where their stem cells were first collected for gene editing.
The trial was conducted as part of the multi-centre 'Ruby Trial'.
He received chemotherapy to clear out the remaining bone marrow, making room for the repaired cells, which were then injected back into his body.
The treatment was well tolerated and no serious side effects were reported. After treatment, all patients successfully recovered their white blood cells and platelets.
The researchers noted that all patients have been free of painful events since treatment, and resolution of anemia was seen in those treated for five months or longer.
"It is encouraging that this gene-editing treatment is showing promising efficacy for sickle cell patients," said Rabie Hanna, MD, MD, PhD, of Cleveland Clinic Children's and presenting investigator of the RUBY trial.
Sickle cell disease is a genetic blood disorder that causes red blood cells to become sickle-shaped.
In sickle cell disease, abnormal cells block blood flow and break down easily, causing problems like severe pain, liver and heart problems, and a shorter life span, usually in the mid-40s.
18 patients, two of whom were treated at Cleveland Clinic Children's in the US, underwent a procedure where their stem cells were first collected for gene editing.
The trial was conducted as part of the multi-centre 'Ruby Trial'.
He received chemotherapy to clear out the remaining bone marrow, making room for the repaired cells, which were then injected back into his body.
The treatment was well tolerated and no serious side effects were reported. After treatment, all patients successfully recovered their white blood cells and platelets.
The researchers noted that all patients have been free of painful events since treatment, and resolution of anemia was seen in those treated for five months or longer.
"It is encouraging that this gene-editing treatment is showing promising efficacy for sickle cell patients," said Rabie Hanna, MD, MD, PhD, of Cleveland Clinic Children's and presenting investigator of the RUBY trial.
Sickle cell disease is a genetic blood disorder that causes red blood cells to become sickle-shaped.
In sickle cell disease, abnormal cells block blood flow and break down easily, causing problems like severe pain, liver and heart problems, and a shorter life span, usually in the mid-40s.
