Is CRISPR meant to increase, replace, or remove a specific gene function in sickle cell anemia
Added by Charles C.
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Sickle cell anemia is a genetic disorder caused by a mutation in the HBB gene, which encodes the beta-globin subunit of hemoglobin. This mutation leads to the production of abnormal hemoglobin, causing red blood cells to become rigid and sickle-shaped. Show more…
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crispr cas 9 in sickel cell animia
Asma V.
Explain how to use the powerful gene editing technology of CRISPR-Cas9 system to potentially change the mutated gene (causing sickle-cell disease) back to a normal gene.
Adi S.
Part C One medical condition showing promise with CRISPR-Cas9 treatment is sickle-cell disease. A mutation in the HBB gene, which codes for beta-globin, causes abnormal hemoglobin to be produced in blood cells. This distorts red blood cells into an abnormal sickle shape and causes clumping, leading to pain and serious medical complications. Scientists would like to repair the mutant HBB gene in the bone marrow of sickle-cell disease patients so that normal hemoglobin can be produced in their blood cells. To use CRISPR-Cas9 in this application, some parts of the technique will be specific to sickle-cell treatment and some parts will be the same as other CRISPR-Cas9 uses. How would the CRISPR-Cas9 system have to be customized for this application? Select all that apply. The guide RNA would have to target the section of the HBB gene with the mutation. Bone marrow cells would have to be targeted for modifications. The Cas9 protein would have to be altered to make double-strand DNA breaks. A normal copy of the HBB gene would have to be provided. Unique repair enzymes would have to be inserted into the cells.
Jennifer S.
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