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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Bacteria has a hard time getting inside a dry egg. Washing eggs removes the bloom and allows bacteria into the egg. Washing eggs in cool water pulls bacteria in even faster
Hmm, let me see. This illustration appears to be an image of a cell. But, there aren't any answers that has cell. Using logic, it would be an organelle. Mainly because there aren't any arrows directing to a specific organelle. Since a cell wall is an organelle, it has to be D.
Answer:
Which of the following factors contributes to the distribution of biodiversity on Earth?
Inter-species interaction contribute greatly to the distribution of biodiversity on earth as it gives room for migration which helps to increase biodiversity
Explanation:
In frogs, different mating seasons are a barrier to reproduction.
