Sickle cell anemia is a recessive disorder in which hemoglobin loses the ability to effectively deliver...

Sickle cell anemia is a recessive disorder in which hemoglobin loses the ability to effectively deliver oxygen to the tissues. Gene therapy in which hemoglobin gamma-chain is introduced to sickle cell anemia patients seems to be a promising new therapy to correct the disease. Why do you think this is possible, based on your knowledge of the sickle cell mutation and the different hemoglobin polypeptide chains?

Expert Solution

sickle cell disease is a inherited blood disease due to defective hemoglobin where red blood cells (RBC) are not shaped in their usual way. it is a recessive disorder.

however, gene therapy is a promising way to diagnose this disease.

stem cell transplant is the known method to cure this disease.

now as for gene therapy;-

In the fetus, hemoglobin genes (HBG1 and HBG2) are present,

two hemoglobin alpha chains together with two hemoglobin gamma chains will constitute fetal hemoglobin (HBF).

After birth, the switch to adult hemoglobin (HBA) takes place, with only a tiny fraction of HBF stays back in adults.

The normal adult tetramer consists of two hemoglobin alpha and hemoglobin beta chains.

Mutations in the hemoglobin beta gene causes sickle cell anemia. so, by mutations the HBG that after the switch from HBF (using the gamma gene) to HBA (using the beta gene) becomes apparent.

so the fetal form complex could possibly solve or decrease the sickle disease.

gladiator answered 1 year ago

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