Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia
, and Myelofibrosis (MF)
Summary: TEHRAN (FNA)- Researchers have used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several blood disorders, including sickle cell disease and beta-thalassemia
syndrome (BTS) is a group of hereditary blood disorders characterised by genetic mutations in [beta]-globin chain genes resulting in reduced or absent [beta]-globin chain synthesis.
a leading genome editing company, announced results from a follow-up study to assess two different CRISPR genome editing strategies, one targeting the BCL11A erythroid enhancer (BC11Ae) and one targeting the beta-globin locus, for the treatment of sickle cell disease and beta-thalassemia
. The Company reported the data at the 24th Congress of the European Hematology Association in Amsterdam.
All over world, there are more than 250 million (1.5% of world populations) carriers of beta-thalassemia
gene, and in south East Asia, there are 40 million carrier of gene.
Editas Medicine announced results from a follow-up study to assess two different CRISPR genome editing strategies, one targeting the BCL11A erythroid enhancer and one targeting the beta-globin locus, for the treatment of sickle cell disease and beta-thalassemia
. In this study, NBSGW mice received an infusion of human CD34+ cells which had been edited either at the BCL11Ae or at the beta-globin locus.
A Phase 3 trial (COMMANDS) in ESA-naive, lower-risk MDS patients, the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia
, and a Phase 2 trial in myelofibrosis are ongoing.
Metabolic and endocrinologic complications in beta-thalassemia
major: a multicenter study in Tehran.
Sickle Cell, SCIDs, beta-Thalassemia
, cancer); (c) shortened testing time for challenging antibody workups; (d) saving costs in laboratory testing, frequency of transfusions, and hospital bed stay; (e) saving in future testing costs as the genotype never changes (except for in bone marrow transplants); (f) bypassing common limitations in serology; (g) matching blood unit to patient at the genotype level to account for more blood group variability, and (h) improving transfusion outcomes for patients in need.
Thalassemia is one of the most common inherited blood disorders in the world.1 Worldwide 240 million heterozygous and 200,000 homozygous thalassemic children are born annually.2 It is estimated that about 3-5% of the world's population carries gene for beta-thalassemia
trait.3 Highest frequency of carrier for [beta] thalassemia is reported in Southeast Asia.4 According to WHO thalassemia is spreading steadily with varying prevalence rate in different regions.
BLA submission includes both myelodysplastic syndromes and beta-thalassemia
"Combining gene editing with an autologous stem-cell transplant could be a therapy for sickle-cell disease, beta-thalassemia
and other blood disorders."