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Once inside the body the vector introduces the therapeutic gene into host cells, and the protein encoded by the newly inserted gene is then produced by the body's own cells. [11] This type of therapy can correct for the missing protein/enzyme in patients with lysosomal storage diseases. [1]
A feedback loop involving erythropoietin helps regulate the process of erythropoiesis so that, in non-disease states, the production of red blood cells is equal to the destruction of red blood cells and the red blood cell number is sufficient to sustain adequate tissue oxygen levels but not so high as to cause sludging, thrombosis, or stroke ...
Erythropoietin (/ ɪ ˌ r ɪ θ r oʊ ˈ p ɔɪ. ɪ t ɪ n,-r ə-,-p ɔɪ ˈ ɛ t ɪ n,-ˈ iː t ɪ n /; [1] [2] [3] EPO), also known as erythropoetin, haematopoietin, or haemopoietin, is a glycoprotein cytokine secreted mainly by the kidneys in response to cellular hypoxia; it stimulates red blood cell production (erythropoiesis) in the bone marrow.
GDEPT is a suicide gene therapy in which the enzyme required for prodrug conversion is produced within the target cell, using a gene delivered to it by gene therapy. When an adequate differential exists between the targeted cell and endogenous tissue, non-toxic prodrug is administered and is subsequently converted into its toxic form within the ...
Many human blood cells, such as red blood cells (RBCs), immune cells, and even platelets all originate from the same progenitor cell, the hematopoietic stem cell (HSC). [1] As these cells are short-lived, there needs to be a steady turnover of new blood cells and the maintenance of an HSC pool. This is broadly termed hematopoiesis. [2]
A defect of the enzyme results in the premature breakdown of red blood cells. This destruction of red blood cells is called hemolysis. [6] Red blood cell breakdown may be triggered by infections, certain medication, stress, or foods such as fava beans. [1] [3] Depending on the specific mutation the severity of the condition may vary. [2]
Erythropoiesis-stimulating agents (ESA) are medications which stimulate the bone marrow to make red blood cells. [1] They are used to treat anemia due to end stage kidney disease, chemotherapy, major surgery, or certain treatments in HIV/AIDS. [1] [2] In these situations they decrease the need for blood transfusions. [2]
Hematology (spelled haematology in British English) is the branch of medicine concerned with the study of the cause, prognosis, treatment, and prevention of diseases related to blood. [ 1 ] [ 2 ] It involves treating diseases that affect the production of blood and its components, such as blood cells , hemoglobin , blood proteins , bone marrow ...