Thalassemia Stem Cell Therapy Development

A group of inherited hemolytic anemia disorders is known as thalassemia. Because of disease-causing mutations in the - or -globin genes, thalassemia is characterized by the impaired synthesis of globin chains. The type, quantity, and clinical symptoms of the deficient nucleoprotein chains are highly variable due to the complexity and diversity of genetic defects. CD BioSciences has launched a development service for thalassemia stem cell therapies, dedicated to advancing the treatment of this disease.

Overview of Thalassemia

A type of hereditary hemoglobin disorder called thalassemia is brought on by a disorder of globin synthesis. Depending on the severity of the anemia, it can be divided into light, intermediate and heavy types. The disease typically has a recessive chromosomal inheritance. The specific type of globin chain(s) whose synthesis is impaired distinguishes the different types of thalassemia, including α-, β-, δβ-, γδβ-, δ-, and γ-thalassemia. Hematopoietic stem cell transplantation is currently the only clinical cure for beta-thalassemia major. However, further molecular mechanisms need to be understood and the method needs to be perfected for real clinical application.

Molecular Basis of Thalassemia

Proteins with symmetric pairings of α-like and β-like globin dimers make up human inherited hemoglobin. Three functional globin genes, the embryonic ζ gene (HBZ) and two fetal/adult α, α1 and α2, genes (HBA1 and HBA2), comprise the α-globin genome. Five functional globin genes, the embryonic ε gene (HBE), two fetal Gγ and Aγ genes (HBG2 and HBG1), and adult δ and β (HBD and HBB) genes, comprise the β-globin genome.

The human α-globin gene cluster and the β-globin gene cluster. (Jee-Soo Lee. et al. 2021)

• Molecular Basis of α-thalassemia
  Deletions involving one or more α-globin genes are the main cause of α-thalassemia. Due to the duplication and localization of α-globin genes into two highly homologous units, gene deletion is most likely caused by an unequal crossover between these units during meiosis. Additionally, non-deletional variants of thalassemia result in a greater decrease in α-globin chain expression than the form of the condition in which only one α-globin gene is deleted.
• Molecular Basis of β-thalassemia
  Contrary to α-thalassemia, which is primarily caused by deletions, the majority of β-thalassemia-causing variants are non-deletions, such as short insertion or deletions that cause frameshift and single nucleotide substitutions.

Advances in Stem Cell Therapy for Thalassemia

Hematopoietic stem cell transplantation is the only comprehensive approach with the potential to treat thalassemia to date. Substantial progress has been made with allogeneic hematopoietic stem cell transplantation, and current clinical trials are investigating new conditional regimens. A better understanding of the underlying mechanisms of the disease could improve existing therapeutic approaches and the discovery of new treatment options.

Our Services

CD BioSciences offers thalassemia stem cell therapy development services. We have a mature experimental process and professional experimental skills. Based on our professional laboratory team, we can contribute to the development of stem cell therapy for thalassemia.

As a pioneer in biotechnology, CD BioSciences has grown into one of the largest independent biotechnology companies in the world. CD BioSciences is committed to providing professional and efficient service to our customers around the world. If you are interested in our service, please contact us.

Reference

1. Jee-Soo Lee. et al. (2021). Molecular basis and diagnosis of thalassemia. Blood Res. 56(S1): S39-S43.