Pemphigus
Pemphigus is a collection of autoimmune skin disorders characterized by the formation of blisters, sores, or fluid-filled lesions on the skin and mucous membranes. Our company, leveraging cutting-edge biotechnological advancements and comprehensive research capabilities, is exceptionally well-equipped to address your specific needs in drug and therapy development for pemphigus.
Introduction to Pemphigus
Pemphigus is a rare autoimmune disorder predominantly affecting the skin and mucous membranes, where autoantibodies target proteins essential for cell adhesion, leading to blister formation. Recent literature highlights an incidence rate ranging from 0.1 to 0.5 cases per 100,000 people annually, varying significantly by geographic location and ethnicity.
Pathogenesis of Pemphigus
The pathogenesis of pemphigus involves a complex autoimmune process primarily mediated by pathogenic autoantibodies directed against desmosomal adhesion proteins, specifically desmoglein 1 (Dsg1) and desmoglein 3 (Dsg3). These proteins are crucial for the adhesion of keratinocytes in the epidermis and mucosal surfaces. In pemphigus, the binding of these autoantibodies to Dsg proteins disrupts keratinocyte adhesion, leading to the characteristic blistering of skin and mucosa seen in the disease. This process is termed acantholysis.
Fig. 1 Pathogenic effects of pemphigus autoantibodies from clinical and experimental findings. (Schmidt, E., et al., 2019)Diagnosis Development of Pemphigus
- Deeper understanding of pemphigus pathophysiology led to the development of direct and indirect immunofluorescence techniques. Direct immunofluorescence (DIF) involves biopsy of the affected skin or mucosa and is used to detect immunoglobulin G (IgG) and complement component 3 (C3) deposited in a net-like pattern around the cells in the epidermis.
- For more precise diagnosis, ELISA (enzyme-linked immunosorbent assay) tests were developed to detect antibodies against desmoglein 1 and desmoglein 3, the specific proteins targeted by the autoantibodies in pemphigus vulgaris and pemphigus foliaceus.
Therapy Development of Pemphigus
Small molecule drugs focus on inhibiting specific pathways involved in the autoimmune process of pemphigus. An example is Rituximab. It targets CD20 on B cells, reducing the production of pathogenic antibodies.
Gene therapies for pemphigus are still in the exploratory stages but hold great promise for providing long-lasting or permanent solutions to the underlying genetic triggers of the disease. Potential strategies include gene editing tools like CRISPR/Cas9 to modify specific immune cells to prevent them from attacking healthy tissue.
Cell therapies offer a novel approach to treating pemphigus by using cells as therapeutic agents. One promising area is the use of regulatory T cells (Tregs) to enhance immune tolerance. Since pemphigus is an autoimmune disorder, increasing the population of Tregs can help modulate the immune response against skin cells.
Monoclonal antibodies (mAbs) represent a cornerstone in pemphigus therapy by targeting specific immune cells or proteins involved in the disease's pathology. Rituximab, as mentioned earlier, is widely used and approved for pemphigus therapy. It depletes CD20+ B cells, which are crucial in the pathogenesis of the disease.
Our Services
Our company adopts a partnership-driven approach. We collaborate closely with clients to craft tailored, innovative Pemphigus therapy strategies and ensure robust support throughout the process.
Platforms of Pemphigus Therapy Development
Animal Models of Pemphigus
We have established expertise in developing and utilizing relevant animal models that closely mimic the disease characteristics and response to therapy. These models enable us to evaluate the safety and efficacy of potential therapies.
| Non-Genetically Engineering Models | ||
| We provide diverse model choices customized to meet specific research needs related to Pemphigus. These models allow researchers to simulate and study the complex biological processes associated with Pemphigus. | ||
| Induced Disease Models | ||
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| Xenograft Models | ||
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| Genetically Engineered Models | ||
| Our expertise in genetic engineering techniques, such as CRISPR/Cas9 technology, allows us to generate accurate and reliable models that recapitulate the genetic alterations observed in human Pemphigus. | ||
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| Optional Species | Mice, Rats, Non-human primates, Others | |
In addition to these models, our comprehensive services encompass other models that target specific signaling pathways and molecular targets.
If our services align with your goals, please contact us for more details.
References
- Schmidt, E., et al., "Pemphigus." Lancet, (2019). 394(10201): p. 882-894.
- Ramani, P., et al., "Apoptolysis: a less understood concept in the pathogenesis of Pemphigus Vulgaris." Apoptosis, (2022). 27(5-6): p. 322-328.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.