You land a post as scientific investigator with a pharmaceutical company that would like to develop drugs to treat people with sickle-cell anemia. They want ideas from you! What molecular properties of Hb S might you suggest as potential targets of drug therapy?

Short Answer

Expert verified
Potential targets of drug therapy for sickle cell anemia could focus on preventing the polymerization of deoxygenated Hb S, raising oxygen affinity of Hb S to deter sickling conditions, or promoting production of Hb F to reduce the presence of Hb S and its subsequent sickling.

Step by step solution

01

Understand the Disease & Properties of Hb S

Sickle cell anemia is a severe hereditary form of anemia in which a mutated form of hemoglobin distorts the red blood cells into a crescent shape at low oxygen levels. Hb S carries an abnormal form of beta-globin due to a genetic mutation. The properties to focus on include its low oxygen affinity, the tendency for deoxygenated Hb S to polymerize, and how these conditions cause cells to sickle.
02

Identify Potential Targets Formulated from Properties

Following the understanding of the properties from step 1, potential targets include: 1. Preventing the polymerization of deoxygenated Hb S, as this is the primary cause of sickling. 2. Modifying the oxygen affinity of Hb S, so that it retains more oxygen, preventing the deoxygenation condition that leads to its polymerization. 3. Stimulating the production of fetal hemoglobin (Hb F) which does not sickle and can stop the sickling process.
03

Suggest Drug Therapy Targets

The drug therapies should ideally target: 1. Prevention or reduction of Hb S polymerization. Drugs can be developed to interrupt the bonding process that enables this polymerization. 2. Modification of Hb S's oxygen affinity. Drugs can be sought which can increase the oxygen-holding capacity of the Hb S. 3. Stimulation of Hb F production. Certain agents/drugs are known for their ability to stimulate Hb F production, thus reducing the relative concentration of Hb S, decreasing sickling.

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