SCA is a common monogenic blood disorder with potentially devastating consequences due to chronic and episodic disease; it has a massive impact on the health-care system and is linked to a significant reduction in life span.
We show that gene therapy with a lentivirus vector expressing γ-globin has the potential for a one-time cure and define the parameters required to cure the disease. We also show a preclinical in vivo method for determining the minimal amount of genetically corrected hematopoietic stem cells needed to correct disease, which is important in the design of clinical gene therapy trials.
The expression of the γ-globin gene in hematopoietic stem cells (HSCs) results in enough postnatal fetal hemoglobin (HbF) to correct sickle cell anemia (SCA) in the Berkeley "humanized" sickle mouse. We assessed critical parameters for correction after de-escalating the number of transduced HSCs in transplant recipients using reduced-intensity conditioning and varying gene transfer efficiency and vector copy number.
The minimal amount of HbF, F cells, HbF/F-cell, and gene-modified HSCs required for correcting the sickle phenotype was determined using a systematic quantification of functional and hematologic red blood cell (RBC) indices, organ pathology, and life span.
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Answer:
A. delivery of oxygen to tissue cells
Explanation:
Blood is a liquid tissue formed by different types of cells suspended in plasma. It circulates throughout our body, through veins and arteries. Veins carry blood from the organs and tissues to the heart, while arteries carry blood from the heart to the organs and tissues.
One of the basic functions of the blood is the transport of substances, of which the transport of oxygen to the tissues, the transport of oxygen and nutrients to the cells, the removal of tissues from cellular activities (such as carbon dioxide produced in the cell). cellular respiration and the conduction of hormones by the body.
Controls what goes in and out of a cell
