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Gene Therapy For Hemophilia A

Gene Therapy For Hemophilia A:

The Union Minister of Science & Technology addressing the National Science Day 2024 programme stated that India conducted its first human clinical trial of gene therapy for hemophilia A (FVIII deficiency) at Christian Medical College (CMC) Vellore.

  • Hemophilia is a group of rare bleeding disorders caused by a congenital deficiency in specific clotting factors.
  • The most prevalent form is Hemophilia A.
  • Hemophilia A results from a deficiency in a crucial blood clotting protein known as factor VIII.
  • Due to this deficiency, individuals experience prolonged bleeding after injuries, as their blood takes longer to clot than usual.
  • It is primarily inherited (genetic) and follows an X-linked recessive pattern, meaning the gene responsible for factor VIII production is located on the X chromosome.
  • Males have one X and one Y chromosome, while females have two X chromosomes.
  • If a male inherits an X chromosome with the defective gene from his mother, he will have hemophilia A.
  • Females with one defective copy typically do not experience symptoms because the other X chromosome usually provides enough factor VIII.
  • However, females can have hemophilia A if they inherit two defective copies, one from each parent (much less common).
  • Symptoms: The severity of hemophilia A varies depending on the level of factor VIII activity in the blood
  • The treatment involves replacing the missing blood clotting factor so that the blood can clot properly.
  • This is typically done by injecting treatment products, called clotting factor concentrates, into a person’s vein.
  • The two main types of clotting factor concentrates available are:
    • Plasma-derived Factor Concentrates: Derived from human plasma, which is the liquid component of blood containing various proteins, including clotting factors.
    • Recombinant Factor Concentrates: Introduced in 1992, recombinant factor concentrates are genetically engineered using DNA technology and do not rely on human plasma.
    • They are free from plasma or albumin, eliminating the risk of transmitting bloodborne viruses.