CTX001 is an autologous therapy, wherein a patient’s own cells are edited and used for the treatment. Hematopoetic stem cells were surgically extracted from the bone marrow of trial participants. Then, CRISPR was used to edit the BCL11A gene, repressor of fetal hemoglobin (Hbf) expression,...
CTX001 is an ‘ex vivo’ application of gene-editing, in which the technology is used to modify a patient’s own cells outside the body. It uses CRISPR/Cas9 to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells, by introducing...
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. H...
CTX001 is an investigational ex vivo CRISPR gene-edited therapy for patients suffering from ß-thalassemia or sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of...
Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells...
CTX001 is an investigationalex vivoCRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin...
Cytoskeletal changes of human umbilical vein endothelial cells (HUVECs) treated with 10 uM KML001 were assessed by immune blotting and confocal imaging. KML001 degraded tubulin protein in HUVECs, which may be related to vascula...
Keywords: dye-sensitized solar cells ; TCO-free ; flexible ; plastic substrates ; transfer methoddoi:10.1021/acsnano.5b01346Kicheon YooJae-Yup KimJin Ah LeeJin Soo KimMin Jae Ko