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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Microvilli which line down the luminal surface of those epithelial cells represent the resorptive function of the proximal tubule.
<span>The high quantity of mitochondria inside the epithelial cells of the proximal convoluted tubule is needed in order to supply the energy for the active transport of sodium ions (to enter the cell from the luminal side).</span>
The answers are
B. large grain sizes C. more precipitation D. warmer temperatures
Answer:
igneous rock
Explanation:
Look at the chart metamorphic rock melts turning into magma magma solidifies into IGNEOUS ROCK
