Memorial Sloan Kettering Researcher Recognized for Stem Cell Therapy Trial for Thalassemia Patients | Memorial Sloan Kettering...
It is characterized by the inability of red blood cells to make a protein called beta-globin. In sickle cell disease, a related ... disorder affecting the same gene, the red blood cells make an abnormal form of beta-globin. ...https://www.mskcc.org/announcements/mskcc-researcher-recognized-stem-cell-therapy-trial-thalassemia-patients
Cell-Based Therapies Show Promise | Memorial Sloan Kettering Cancer Center
The disease is characterized by the inability of red blood cells to make a protein called β-globin. In a related disorder ... The β-globin protein is part of hemoglobin, a vital protein that delivers oxygen throughout the body. ... modified to contain the functional globin gene, and given back to the patients. The hematopoietic stem cells can come from the ... the body's natural production of blood cells prior to the reinfusion of the stem cells engineered with the normal globin gene. ...https://www.mskcc.org/blog/cell-based-therapies-show-promise
The Michel Sadelain Lab | Sloan Kettering Institute
... focus on the role of locus control regions and insulators in regulating the transcription of randomly integrated beta globin ...https://www.mskcc.org/research/ski/labs/michel-sadelain/overview
Globin Gene Therapy | Memorial Sloan Kettering Cancer Center
MSK's innovative globin gene transfer therapy offers the potential to free patients from a lifetime of red blood cell ... which is believed to achieve better expression of β-globin on a per-copy basis, and therefore the potential for superior ... utilizing a lentiviral vector to stably insert a functional β-globin gene, and then infusing the patient with engineered HSCs ...https://www.mskcc.org/research-advantage/support/technology/available/globin-gene-therapy
Synapse - Depletion of L3MBTL1 promotes the erythroid differentiation of human hematopoietic progenitor cells: Possible role in...
The Pc G Protein L3 Mbtl1 Transcriptionally Represses Human Embryonic And Fetal Globin Genes: A Novel Prospect For Hb F ...https://synapse.mskcc.org/synapse/works/4844