Hemophilia
Hemophilia is a rare genetic blood disorder. With our pioneering efforts in hemophilia research, we are at the forefront of developing cutting-edge diagnostic tools and therapeutics to facilitate the effective management of hemophilia. As your reliable partner in hemophilia research, we offer unmatched support to fulfill your scientific research needs.
Overview of Hemophilia
Hemophilia is a rare genetic blood disorder that causes reduced blood clotting, leading to an increased risk of bleeding or bruising. Hemophilia encompasses two primary types: hemophilia A and hemophilia B. These forms of hemophilia are inherited in an X-linked recessive pattern. As a result, the diseases predominantly affect males.
| Disease Name | Pathogenesis | Incidence (Male Births) | Inheritance |
|---|---|---|---|
| Hemophilia A | Low levels of clotting factor VIII (FVIII) | 1/5,000–1/10,000 | X-linked recessive inheritance |
| Hemophilia B | Low levels of clotting factor IX (FIX) | 1/40,000 | X-linked recessive inheritance |
Fig. 1 Mechanisms of anti-FVIII alloantibody development. (Prezotti, et al., 2022)Diagnostic Progress of Hemophilia
Progress in laboratory testing and molecular diagnostics has greatly contributed to the development of hemophilia diagnosis.
- Complete Blood Count (CBC)
- Activated Partial Thromboplastin Time Test
- Genetic Testing
- Chorionic Villus Sampling (CVS) Examination
- Prothrombin Time (PT) Test
- Clotting Factor Test
- Next-generation Sequencing (NGS)
- Amniocentesis
Types of Hemophilia Therapy
Clotting Factors Replacement Therapy
One of the main goals in the development of therapeutics for hemophilia is to replace missing or deficient clotting factors. The production of recombinant coagulation factors by genetic engineering has revolutionized the therapeutics of hemophilia. Recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) have become standard therapeutics for individuals with hemophilia A and hemophilia B, respectively.
Gene Therapy
Gene therapy has emerged as a promising approach for treating hemophilia. It involves the introduction of functional copies of the defective gene responsible for hemophilia into the cells of hemophilia individuals. The goal is to enable the production of sufficient clotting factors to restore normal blood clotting. Gene therapy offers the potential for long-lasting effects and reduced reliance on frequent infusions of clotting factors.
Our Services
Drawing upon our deep expertise in biotechnology and extensive experience in the industry, our company offers all-encompassing solutions for diagnostic and therapeutic research dedicated to hemophilia.
- Hemophilia Diagnostic Development Services: For rare genetic diseases such as hemophilia, our company offers diagnostic development services and state-of-the-art next-generation sequencing (NGS) products. We are dedicated to assisting you in the development of rapid and point-of-care diagnostic tests for hemophilia, ensuring accurate and timely detection.
- Hemophilia 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. Specifically, we excel in utilizing gene editing technologies like CRISPR/Cas9 to investigate gene therapy for hemophilia, which holds tremendous promise as a future therapeutic approach.
- Hemophilia Animal Model Development Service: To support the preclinical research and development of hemophilia therapeutics, we offer hemophilia animal models development services to facilitate your pharmacokinetics study and drug safety evaluation.
| Genetically Engineered Models | ||
| Our company is dedicated to the development of genetically engineered hemophilia models. Our team of scientists utilizes techniques such as transgenics and gene editing to introduce or modify specific genes in various animal models, effectively inducing the hemophilia phenotype. | ||
| Optional Models |
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| Optional Species | Mice, Dogs, Rats, Pigs, Sheep, Pigs, 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
- Prezotti, Alessandra NL, et al. "Immunogenicity of current and new therapies for hemophilia a." Pharmaceuticals 15.8 (2022): 911.
- Yen, Ching-Tzu, et al. "Current animal models of hemophilia: the state of the art." Thrombosis journal 14 (2016): 101-106.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
