Sickle Cell Disease (SCD)
Sickle cell disease (SCD) is a group of rare blood disorders. With our company's profound expertise in SCD research, we are well-equipped to offer tailored solutions and comprehensive support to facilitate your research process from SCD therapy development to therapy commercialization.
Overview of SCD
SCD is a group of uncommon hematological disorders characterized by the presence of abnormal sickle-shaped red blood cells. These abnormal cells can result in various complications and health issues. SCD affects approximately 100,000 Americans. It affects about 1 in 365 births among African Americans and occurs in approximately 1 in 16,300 births among Hispanics.
Fig. 1 Schematic pathophysiology review of sickle cell disease and its main different targets for intervention. (Salinas Cisneros, et al., 2020)Pathogenesis of SCD
The occurrence of SCD is related to mutations in the β-globin gene. This mutation results in the production of abnormal hemoglobin (HbS), causing red blood cells to become rigid and take on a sickle shape. These sickle cells tend to clump together, obstructing blood flow and leading to tissue damage. Additionally, these cells have a shorter lifespan than normal red blood cells, causing chronic anemia.
Diagnostic Methods of SCD
Accurate and timely diagnostics play a crucial role in managing SCD. Over the years, significant advancements have been made in diagnostic techniques for SCD. The following are common diagnostic methods for SCD.
- Hemoglobin Electrophoresis
- Complete Blood Count (CBC)
- Sickle Solubility Test
- Hemoglobin High-Performance Liquid Chromatography
- Genetic Testing
- New Generation Sequencing (NGS)
Targets of SCD Therapy
Fetal Hemoglobin (HbF)
Increased HbF production in SCD inhibits the polymerization of sickle hemoglobin (HbS) and reduces sickling of red blood cells. Hydroxyurea increases HbF production, reduces the frequency of painful crises in individuals with SCD and reduces the need for blood transfusions. It is FDA-approved for the therapeutics of SCD.
Adhesion of Sickle Cells to Endothelial Cells
Adhesion of sickle cells to endothelial cells causes vascular occlusion and tissue damage in SCD. Crizanlizumab targets sickle cell adhesion to endothelial cells by inhibiting the binding of P-selectin to P-selectin glycoprotein ligand-1 (PSGL-1), thereby reducing the frequency of vaso-occlusive crises in SCD individuals.
Our Services
As a frontrunner in the field of biological research and CRO services, our company is dedicated to offering holistic solutions for the diagnostics development and therapy research of SCD. Through collaborations with industry experts, we have established all-encompassing platforms for rare diseases to facilitate SCD therapy development.
Platforms of SCD Therapy Development
Recognizing the significance of dependable animal models in SCD disease research, our company is committed to offering animal model development services that facilitate preclinical research and aid in drug discovery endeavors.
Animal Models of SCD
| Genetically Engineered Models | ||
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| Our company is dedicated to the development of genetic engineering models for SCD. Our scientists employ techniques such as pronuclear microinjection or gene targeting to introduce the human sickle hemoglobin gene (HbS) into the mouse genome. This genetic modification prompts the mice to produce sickle-shaped red blood cells, mimicking the characteristics of SCD. | ||
| Optional Models |
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| Induced Models | ||
| To induce SCD-like symptoms in animal models, various approaches can be utilized. For example, our scientists subjected mice to hypoxia or dehydration to trigger the development of red blood cell sickling and vaso-occlusive crises. In the baboon model, sickle-like lesions and SCD-like complications were induced by administering deoxygenating agents or exposing them to hypoxia. | ||
| Optional Species | Mice, Zebrafish, Pigs, Non-human Primates (Baboons and Rhesus Macaques), Others | |
With complete animal species resources, we can meet your diversified preclinical research including drug safety evaluation and pharmacokinetic analysis. If you are interested in our services, please feel free to contact us for more details and quotation information for related services.
References
- Salinas Cisneros, Gabriel, and Swee L. Thein. "Recent advances in the treatment of sickle cell disease." Frontiers in Physiology 11 (2020): 513588.
- 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.