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:
ATP synthase donates an electron to the electron transport chain.
DNA- Deoxyribonucleic Acid is a molecule composed of two chains or double strands that coil around each other forming a double helix to carry the genetic information for the development of an organism.
<u>Explanation:</u>
- DNA contains the information that instructs a living organism to develop, grow, survive and reproduce.
- The information instructed is found inside every cell and is passed down from parents to their children.
- DNA is made of nucleotide molecules which in turn contains a phosphate group, a sugar group, and a nitrogen base.
- Deoxyribose in DNA is another form modified from ribose sugar.
On the laboratory bench right in front of you, there is a bone. You can tell that it has an articular surface that is smooth and just slightly concave. This skeletal feature would be referred to as a Facet.
<h3>What is the composition of human bone?</h3>
A transcription factor collagen forms the structure of bones, and calcium phosphate, a mineral, gives the framework strength and hardness. When other body parts require calcium, bones release some of the calcium they have stored into the blood.
<h3>Why are bones so durable?</h3>
A living, developing tissue is bone. Collagen, a polypeptide that provides a soft structure, and calcium, a mineral that gives strength and hardness, make up the majority of its composition. Together, these factors give bone the strength and flexibility it needs to withstand stress.
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