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;
-PET scan
A PET scan uses radioactive markers in the blood to monitor blood flow and metabolic activity via x-ray
Explanation;
-A positron emission tomography (PET) scan is an imaging test that helps reveal how the tissues and organs are functioning. A PET scan uses a radioactive drug (tracer) to show this activity. This scan can sometimes detect disease before it shows up on other imaging tests.
-A PET scan is used to diagnose certain health conditions, to plan treatment, to find out how an existing condition is developing, and to see how effective a treatment is.
-In a PET scan, a machine detects radiation that is emitted by a radiotracer. A radiotracer consists of radioactive material that is tagged to a natural chemical, such as glucose.
Answer:
hydorpher i think
Explanation:
sorry if i spelled that wrong :)