Beta-Thalassemia (B-THAL)
Beta-thalassemia (B-THAL) is an inherited blood disorder characterized by reduced levels of functional hemoglobin. With our company's profound expertise in B-THAL research, we are well-equipped to offer tailored solutions and comprehensive support to facilitate your research process from B-THAL therapy development to therapy commercialization.
Overview of B-THAL
B-THAL is an autosomal recessive blood disorder characterized by diminished levels of functional hemoglobin, a crucial protein that transports oxygen within red blood cells. This inherited condition poses a substantial global public health challenge, particularly in regions with high prevalence rates, including the Mediterranean, Middle East, and Southeast Asia. B-THAL is a rare condition, with a projected worldwide annual incidence of 1 in 100,000 individuals.
Fig. 1 Current and future therapies for beta-thalassemia major. (De Dreuzy, et al., 2016)Pathogenesis of B-THAL
B-THAL arises from gene mutations that impact the production of beta-globin chains, a vital part of hemoglobin. These mutations can cause reduced or absent synthesis of beta-globin chains, disrupting the balance between alpha-globin and beta-globin chain production. Consequently, functional hemoglobin formation is impaired, leading to ineffective erythropoiesis and subsequent anemia.
Targets of B-THAL Therapy
Beta-Globin Gene Expression
Enhancing the production of β-globin chains or compensating for their defects are important targets for the development of B-THAL therapies. Hydroxyurea, butyrate derivatives, and histone deacetylase inhibitors are drug candidates currently under investigation for modulating β-globin gene expression.
Iron Metabolism and Chelation
Iron overload is a common complication in individuals receiving regularly transfused B-THAL. It is crucial to manage iron levels and prevent organ damage caused by excess iron deposits. Chelating agents, such as deferoxamine, deferiprone, and deferasirox, help prevent iron overload and its associated complications.
Types of B-THAL Therapy
Hematopoietic Stem Cell Transplantation (HSCT)
HSCT remains the sole therapeutic option for B-THAL. HSCT involves the transplantation of healthy stem cells from a compatible donor into the body of a HSCT individual, replacing the defective stem cells and restoring normal hematopoietic function.
Gene Therapy
Gene therapy is a new therapeutic method for B-THAL. Gene therapy utilizes lentiviral vectors containing human β-globin genes and ankyrin insulators to enhance gene transcription and translation, thereby augmenting the production of β-globin.
Our Services
- B-THAL Diagnostic Development Services: For rare genetic diseases such as B-THAL, our company offers diagnostic development services. We are dedicated to assisting you in developing advanced diagnostic tools for B-THAL, ensuring accurate and timely detection.
- B-THAL Therapeutic Development Services: Our company provides a wide range of services for the development of small molecule drug, cell therapy, gene therapy, therapeutic antibody, therapeutic peptide, and therapeutic protein. We are experienced in utilizing lentiviral vectors for gene therapy.
- B-THAL Animal Model Development Service: To support the preclinical research and development of B-THAL therapeutics, we offer B-THAL animal models development services to facilitate your pharmacokinetics study and drug safety evaluation.
| Genetically Engineered Models | ||
| Genetic engineering technology can introduce B-THAL-related mutations into animal genomes. Our company has obtained animal models of B-THAL through pronuclear injection, embryonic stem cell manipulation, or targeted gene editing technologies such as CRISPR/Cas9 to promote the development of B-THAL therapy. | ||
| Optional Models |
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| Optional Species | Mice, Zebrafish, Sheep, Others | |
No matter what stage of research you are at, we can provide you with corresponding research services. If you are interested in our services, please don't hesitate to contact us for more information and a detailed quotation regarding the specific services you require.
References
- De Dreuzy, Edouard, et al. "Current and future alternative therapies for beta-thalassemia major." Biomedical Journal 39.1 (2016): 24-38.
- McColl, Bradley, and Jim Vadolas. "Animal models of β-hemoglobinopathies: utility and limitations." Journal of Blood Medicine (2016): 263-274.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.