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:
Muscle cells and skin cells look and behave differently because their genes are being expressed differently.
Explanation:
- Genes are the sequence of nucleotide that has all the information about the functioning and characteristics of particular cells.
- All genes are actually present in our liver cell but the thing that matter is the gene is expressed differently for different type of functioning.
- So even having the same gene the cells might look different and behave different, its all about turning the gene "on" and "off".
Answer: Leaves
Explanation:
Photosynthesis takes place in things called chloroplasts. Chloroplasts contain a green substance called chlorophyll. Most of these can be found in the leaves of the plant.
