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Genetic Therapy for Sickle-cell Disease

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Questions and Answers

What is the composition of adult haemoglobin tetramer?

Two α and two γ-globins

Two β and two δ-globins

Two α and two β-globins (correct)

Two β and two γ-globins

During which stage of gestation is fetal hemoglobin prevalent?

Third trimester

Second trimester (correct)

None of the trimesters

First trimester

What is the major cause of sickle cell disease complications in newborns?

Low concentration of fetal hemoglobin (correct)

Presence of adult hemoglobin

Co-inheritance of Hereditary Persistence of Fetal Hemoglobin (HPFH)

Genetic mutations in α-globin genes

What is characteristic of patients with sickle-cell disease who co-inherited Hereditary Persistence of Fetal Hemoglobin?

A benign phenotype Signup and view all the answers

Which region is responsible for the regulation of globin gene clusters?

Locus control region (LCR) Signup and view all the answers

How is Hereditary Persistence of Fetal Hemoglobin typically caused?

By point mutations in the promoters of the γ-globin genes Signup and view all the answers

What happens to fetal hemoglobin after birth?

It is replaced by adult hemoglobin Signup and view all the answers

Why do newborn babies not manifest sickle-cell disease complications until they are around 6 months old?

'Fetal hemoglobin' is prevalent until after birth Signup and view all the answers

Which type of hemoglobin protects against sickle-cell disease?

'Fetal hemoglobin' Signup and view all the answers

What is the impact of Hereditary Persistence of Fetal Hemoglobin (HPFH) on individuals with sickle-cell disease?

'Benign phenotype' Signup and view all the answers