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:
The correct answer is - the more specialized pharyngeal jaws are for one diet, the less adapted they are for the ingestion of other foods
Explanation:
Pharyngeal jaws are also called throat jaws and are specific for the prey. These jaws have strong muscles and plastic or easy-to-modify teeth. If an organism has pharyngeal jaws specialized for a specific diet or prey it will have lesser chances to adapted well according to the new diet. For instance, pharyngeal jaws highly specialized for scale eating will not be able to adapt well if the fish ingests algae.
The uncertainty principle<span>, also known as </span>Heisenberg's uncertainty principle<span>, is any of a variety of mathematical inequalities asserting a fundamental limit to the precision with which certain pairs of physical properties of a particle, known as complementary variables</span>
Answer:
reproduction? not entirely sure what the question is asking for, but i think its reproduction.
Explanation:
