Αποτελέσματα Αναζήτησης
Gene therapy aspires to provide cure for thalassaemia through the manipulation of the genome of haematopoietic stem cells, thus compensating for the inadequate or faulty function of mutated genes.
2 Φεβ 2022 · Patients with transfusion-dependent β-thalassemia, the most severe clinical form of this disorder, receive repeated red-cell transfusions in order to prevent severe anemia and increase...
The goal of cell and gene therapy for thalassemia is thus to achieve transfusion independence without exposing patients to the risks of HSC transplantation from a suboptimally matched donor.
20 Μαρ 2016 · Allogeneic hematopoietic stem cell transplantation has been well established for several decades as gene replacement therapy for patients with thalassemia major, and now offers very high rates of cure for patients who have access to this therapy.
15 Δεκ 2023 · EMA has recommended approval of the first medicine using CRISPR/Cas9, a novel gene-editing technology. Casgevy (exagamglogene autotemcel) is indicated for the treatment of transfusion‑dependent beta thalassemia and severe sickle cell disease in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate ...
This review mainly discusses the gene therapy strategies for the thalassemias, including the use of lentiviral vectors, generation of induced pluripotent stem (iPS) cells, gene targeting, splice-switching and stop codon readthrough.
26 Ιαν 2022 · Adding a functional gene to defective blood stem cells is a successful therapy for patients with severe beta thalassemia, according to an international phase-3 clinical trial led...
