Paroxysmal Nocturnal Hemoglobinuria Stem Cell Therapy Development
Stem cell therapy is a potential treatment option for Paroxysmal Nocturnal Hemoglobinuria (PNH) that involves replacing the patient's bone marrow with healthy stem cells from a donor. Stem cell therapy can be a curative treatment for PNH, as it can eliminate the underlying cause of the disease. CD BioSciences provides stem cell therapy development service for PNH based on our professional team and advanced technology.
Introduction into Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disorder of the blood that is caused by a genetic mutation. It is characterized by the destruction of red blood cells (hemolysis), which can lead to anemia, fatigue, weakness, and other symptoms. PNH is caused by a mutation in the PIG-A gene, which is responsible for the production of certain proteins that anchor other proteins to the surface of red blood cells. This mutation causes the loss of these anchor proteins, leading to the destruction of red blood cells by the body's immune system. PNH typically affects adults and is more common in women than men. Symptoms can vary widely depending on the severity of the disease, but common ones include fatigue, shortness of breath, abdominal pain, and blood clots.
Mechanisms of hemolysis in PNH (Brodsky, Robert A, 2021)
Classification of PNH
The classification of PNH is based on the presence of clinical symptoms and laboratory evidence of hemolysis or thrombosis, signs of BMF such as aplastic anemia (AA), myelodysplastic syndromes (MDS), and cytometric analysis of the clone size of cells affected by the GPI defect.
| Classic PNH, type I | There are no obvious signs of BMF. In a complete blood count (CBC), the number of white blood cells and platelets is usually normal. the GPI(-) cell clone is usually more than 50%. Serum lactate dehydrogenase (LDH) activity and bilirubin concentration may be elevated. |
|---|---|
| Aplastic PNH, type II | Approximately 10% to 50% of GPI(-) cell clones are found, along with mild hemolysis and accompanying laboratory features of leukopenia and/or thrombocytopenia. CBC is usually as follows: hemoglobin <10 g/dl, neutrophils <1 G/l, platelets <80 G/l. /The clinical picture is dominated by BMF symptoms. |
| Subclinical form of PNH, type III | Very small clone of GPI(-) cells (1%-10%) in patients with a diagnosis of MDS or AA and no clinical and laboratory evidence of hemolysis. |
Stem Cell Therapy for PNH
Although suppression of the complement system at various stages is a promising treatment modality for patients with PNH, allogeneic hematopoietic stem cell transplantation (HCT) remains the only treatment option, especially for patients with intractable hemolysis or bone marrow failure.
In addition to bone marrow transplants, researchers are exploring other forms of stem cell therapy for PNH, including the use of mesenchymal stem cells (MSCs) to modulate the immune system and promote tissue repair. Early studies suggest that MSCs may have potential as a novel therapy for PNH, but more research is needed to evaluate their safety and efficacy.
Our Services
CD BioSciences offers stem cell therapy development services for PNH, based on our professional laboratory team, advanced technology and years of experience.
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
- Brodsky, Robert A. "How I treat paroxysmal nocturnal hemoglobinuria." Blood vol. 137,10 (2021): 1304-1309.
For research use only, not for clinical use.
