The researchers used CRISPR-Cas9 gene editing to a remove a piece of genetic code that normally turns off fetal hemoglobin
The Fred Hutch researchers used CRISPR-Cas9 gene editing to a remove a piece of genetic code that normally turns off fetal hemoglobin
The trial will use CRISPR in an attempt to boost production of a different kind of hemoglobin (fetal hemoglobin
) that makes it harder for cells to sickle and stick together, CNN reported.
However, the proportion of fetal hemoglobin
seen in younger infants may explain this.
At 13 weeks, in the 100 mg dose group of Population A, there was an increase in the % of F-cells, which are red blood cells that contain fetal hemoglobin
which often precede rises in total HbF.
At 13 weeks, in the 100 mg dose group of Population A, there was an increase (110%) in the percent of F-cells, which are red blood cells that contain fetal hemoglobin
(HbF) which often precede rises in total HbF.
(2) Moreover, the delivery of highly oxygenated fetal hemoglobin
from the placenta to the baby may have a favorable effect on the infant's health in the early postnatal period.
He believes direct evidence of genome editing, "strong" fetal hemoglobin
response and early signs of transfusion independence bode well for ultimate success and should translate into improved likelihood of success in the larger opportunity of sickle cell disease.
Thalassemia, Genetic disorder, Polymorphism, Fetal hemoglobin
, Transfusion, Gamma-globin.
Hereditary persistence of fetal hemoglobin
(HbF) and acquired conditions like methemoglobinemia are alternative forms of hemoglobinopathy.
Under the company's Phase 1/2 clinical trial protocol, a patient's HSCs are isolated from the blood and the cells then undergo ex vivo gene editing using ZFNs to modify a specific sequence of the BCL11A gene that suppresses fetal hemoglobin
production in erythrocytes.