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: c. produce hormones that help regulate many body functions
Explanation:
The endocrine system helps in the release of hormones and regulating them into bloodstream and these hormones that are released then travel to the cells to perform different functions like growth and development, reproduction, controlling of ones mood, metabolism etc.
Therefore, the correct option is C "produce hormones that help regulate many body functions"
The answer is B) metaphase
theory of continental drift, the world was made up of a single continent through most of geologic time. ... According to the generally acceptedplate-tectonics theory<span>, scientists believe that Earth's surface is broken into a number of shifting slabs or </span>plates<span>, which average about 50 miles in thickness....</span>
