Bone Marrow Failure Syndromes Stem Cell Therapy Development
Bone Marrow Failure Syndromes (BMFS) are a group of rare disorders characterized by a decrease in the production of blood cells by the bone marrow. The bone marrow is the spongy tissue found inside bones that produces red blood cells, white blood cells, and platelets. In BMFS, the bone marrow cannot produce enough of one or more of these types of blood cells. CD BioSciences provides stem cell therapy development service for BMFS based on our professional team and advanced technology.
Introduction into Bone Marrow Failure Syndromes
Bone Marrow Failure Syndromes (BMFS) are a diverse group of non-malignant hematological diseases, both inherited and acquired, that result in the reduced production of blood cells in the bone marrow or the bloodstream. This leads to low counts of one or more types of blood cells, even in the absence of other conditions that might affect the bone marrow's function. The cytopenia(s) may affect one or more cell lineages, such as red blood cells, white blood cells, or platelets. It can occur at any age and can be caused by a variety of factors, including genetic mutations, exposure to certain chemicals or drugs, viral infections, and autoimmune disorders. Followings are several types of BMFS:
Aplastic anemia | This occurs when the bone marrow cannot produce enough red blood cells, white blood cells, and platelets. The cause is usually unknown, but it can be caused by exposure to certain drugs, chemicals, or radiation. |
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Diamond-Blackfan anemia | This is a rare genetic disorder that affects the production of red blood cells. |
Fanconi anemia | This is also a genetic disorder that affects the production of all types of blood cells. |
Myelodysplastic syndromes | This is a group of disorders in which the bone marrow produces abnormal blood cells that do not function properly. |
Paroxysmal nocturnal hemoglobinuria | This is a rare disorder in which red blood cells break down prematurely, leading to anemia and other complications. |
Pure red cell aplasia | This occurs when the bone marrow cannot produce enough red blood cells, but the production of white blood cells and platelets is normal. |
The causes of Bone Marrow Failure Syndromes (BMFS) vary depending on the specific type of syndrome. In some cases, BMFS are inherited genetic disorders that result from mutations in genes involved in the production of blood cells. In other cases, BMFS may be acquired and result from exposure to certain chemicals or drugs, viral infections, autoimmune disorders, or other factors that damage the bone marrow.
Schematic of the underlying biology of BMFS (Wegman-Ostrosky, et al., 2017)
Stem Cell Therapy for Bone Marrow Failure Syndromes
Stem cell therapy is a promising treatment option for Bone Marrow Failure Syndromes (BMFS). Stem cells are undifferentiated cells that have the ability to differentiate into various types of cells in the body, including blood cells. In stem cell therapy for BMFS, healthy stem cells are transplanted into the patient's body to replace the faulty cells in the bone marrow that are causing the disease.
Stem cell therapy for BMFS carries certain risks, including graft-versus-host disease (GVHD), in which the donor's immune system attacks the patient's body. However, advances in the field have made the procedure safer and more effective, and it remains a promising option for those with severe BMFS who have exhausted other treatment options.
Our Services
CD BioSciences offers stem cell therapy development services for BMFS, based on our professional laboratory team, advanced technology and years of experience. Our stem cell therapy development service will be offered in BMFS included but not limited as follows:
- Thalassemia
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Reference
- Wegman-Ostrosky, Talia, and Sharon A Savage. "The genomics of inherited bone marrow failure: from mechanism to the clinic." British journal of haematology vol. 177,4 (2017).
For research use only, not for clinical use.