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:
causing them to lose their shelter.
Explanation:
Answer:
T cells and B cells
Explanation:
It is characterized by specificity, immunological memory, and self/nonself recognition. The response involves clonal selection of lymphocytes that respond to a specific antigen. T cells and B cells are the two major components of adaptive immunity.
It is a behavioral adaptation. It is a behavior that a animal does and is used to doing it.
C. is correct because when their bodies are colder they are not able to digest as quickly. they digest much quicker at a warm temperature.
