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 tribute nation is a nation that pays another nation for protection.
Explanation:
In simpler terms, the Big Bang theory says the universe as we know it started with a small singularity, then inflated over the next 13.8 billion years to the cosmos that we know today. In the first second after the universe began, the surrounding temperature was about 10 billion degrees Fahrenheit (5.5 billion Celsius). The cosmos contained a vast array of fundamental particles such as neutrons, electrons and protons. These decayed or combined as the universe got cooler. Over time, however, the free electrons met up with nuclei and created neutral atoms. This allowed light to shine through about 380,000 years after the Big Bang.
The pancreas is connected directly into the small intestines and gallbladder.
Enzymes that are produced by the pancreas are secreted directly into the small
<span>intestine.</span>
