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:
D) AUG
Explanation:
A) false-- AAC codes for Asn
B) false-- AGU codes for Ser
C) false-- UGA is a stop codon
D) true
To read such a table, the one of the right (my preferred choice) , you start by selecting a letter on the left side, then the second letter from the top, and the third from the right side.
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Answer:
The correct answer is: d. loss of blood pressure and loss of oxygen-carrying capacity.
Explanation:
- Blood is a liquid connective tissue which is the major component responsible for transporting nutrients and oxygen to all the cells in the body and for removing carbon dioxide and metabolic wastes from all the cells of the body.
- When there is excessive blood loss, the total volume of blood in the body decreases far below normal. As a result of this the blood pressure (pressure applied by the blood on the walls of the arteries) decreases.
- This is because the rate at which blood is lost is far more than the rate at which it is produced.
- The decrease in blood pressure prevents the blood from travelling easily against the gravity, hence it is unable to reach all the regions of the body, thereby depriving many cells of nutrients and oxygen. The waste products are also not efficiently collected from these cells. This causes the cells in the deprived regions to die.
- Blood carries the most important component required for respiration and metabolism, that is oxygen, in association with haemoglobin molecules. Excessive blood loss causes loss of haemoglobin as well. This can cause reduction in the oxygen-carrying capacity of blood. Hence the cells of the body are unable to carry out metabolic activities properly due to the dearth of oxygen. This causes the death of cells.
- Excessive death of cells causes tissues to become non-functional and that can cause organ failure. Multiple organ failure can pose severe threat to life.
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