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kiruha [24]
2 years ago
14

Perumbeti A, Higashimoto T, Urbinati F, et al. A novel human gamma-globin gene vector for genetic correction of sickle cell anem

ia in a humanized sickle mouse model: critical determinants for successful correction. Blood. 2009;114:1174-1185.
Biology
1 answer:
elena-s [515]2 years ago
5 0

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.

To learn more about sickle cell anemic from the given link

brainly.com/question/17063471

#SPJ4

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Answer:

1) CO₂

2) 0.2551 g

Explanation:

The balanced reactions are:

CaCO₃ + 2HCl → CaCl₂ + H₂O + CO₂

MgCO₃ + 2HCl → MgCl₂ + H₂O + CO₂

1) The gas produced is CO₂.

2) Calculate mass of CaCO₃:

(0.5236 g) (0.4230) = 0.2215 g CaCO₃

Convert to moles:

(0.2215 g CaCO₃) (1 mol / 100.1 g) = 0.002213 mol CaCO₃

Find moles of CaCO₃:

(0.002213 mol CaCO₃) (1 mol CO₂ / mol CaCO₃) = 0.002213 mol CO₂

Convert to mass:

(0.002213 mol CO₂) (44.01 g / mol) = 0.09738 g CO₂

Calculate mass of MgCO₃:

(0.5236 g) (0.5770) = 0.3021 g MgCO₃

Convert to moles:

(0.3021 g MgCO₃) (1 mol / 84.31 g) = 0.003583 mol MgCO₃

Find moles of MgCO₃:

(0.003583 mol MgCO₃) (1 mol CO₂ / mol MgCO₃) = 0.003583 mol CO₂

Convert to mass:

(0.003583 mol CO₂) (44.01 g / mol) = 0.1577 g CO₂

Total mass of CO₂:

0.09738 g CO₂ + 0.1577 g CO₂ = 0.2551 g CO₂

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Answer:

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Explanation:

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