More About inherited abnormalities of bone marrow derived cells
The Institute will have a clinical trial for treating a variety of diseases that are referred to as inherited abnormalities of bone marrow derived cells.There are many conditions included in the inherited abnormalities of bone marrow derived cells clinical trial. inherited abnormalities of bone marrow derived cells can be a red blood cell disorder, other immunodeficiency disorder, or genetic error of metabolism, which is a result of some dysfunction in the blood cells.
Red blood cell disorder - THALESSEMIA
This is a red blood cell disorder that has a deficient beta (B) chain synthesis present during childhood. This deficiency causes the bone marrow to increase cell productivity, which causes a wide range of serious medical complications.Other immunodeficiency disorders
This is a heterogenous group of congenital disorders of the immune system involving B-cells, T-cells, NK cells and Phagocytes. Patients with these disorders are at an increased risk for life-threatening bacterial, fungal and viral infections.These diseases include, but are not limited to:
- Familial hemophagocytic lymphohistiocytosis (FHL)
- Severe combined immunodeficiency disease (SCID)
- Wiskott-Aldrich syndrome (WAS)
- Chediak-Higashi syndrome (CHS)
- Kostmann's syndrome
- Leukocyte adhesion deficiency (LAD)
- Chronic granulomatous disease (CGD)
- Glanzmann's thrombocytopenia
Genetic errors of metabolism
These are enzymatic deficiency conditions, which include many specific diseases. These diseases result in the accumulation and retention of metabolic products, which can lead to progressive neurological deterioration. Treating these diseases with a small amount of normal enzyme might prevent accumulation of the metabolic products, and the resulting progression, degeneration and early death of these individuals. However, genetic therapy for these conditions is not yet available. Therefore, individuals with these diseases are still considered for bone marrow transplantation if they have an appropriate donor. It has also been shown that these patients may benefit from the establishment of chimerism.These diseases include but are not limited to (deficient enzyme is shown):
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- Guacher's Disease (Beta-Glucocerebrosidase)
- Hurler syndrome
- Sanfilippo B
- Fabry disease
- Metachromatic leukodystrophy (arylsulphatase-A)
- Niemann-Pick B (sphingomyelinase)
- Hunter disease (iduronidate-sulphatase)
- Sanfilippo A (heparin-sulphatase)
- Maroteaux-Lamy (arylsulphatase-B)
- Adrenoleukodystrophy (multiple)
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