Gene Therapy: An Effective Treatment Option For Hemophilia?

Hemophilia is a disorder in which the person affected cannot stop bleeding after undergoing an injury. Hemophilia is a rare genetic disorder that leads to the person lacking clotting factors (blood-clotting proteins) in their blood. This causes a disturbance in the natural blood clotting process.

Patients with acute forms of Hemophilia display spontaneous bleeding at their joints. For such patients, internal bleeding in ankles, elbows, knees, elbows, and other joints can be of concern since it can cause tissue and organ damage, making the overall situation potentially life-threatening.

Hemophilia is classified into four types – von Willebrand disease(VIII:vW deficient factor), Hemophilia C(XI deficient factor), Hemophilia B(IX deficient factor), and Hemophilia A(VIII deficient factor).

Historically, there has been no cure for Hemophilia. The current treatment for Hemophilia called Replacement Therapy focuses on maintaining a proper blood clotting process by replacing the missing blood clotting factor. This is done by injecting clotting factor concentrates (treatment products) into the patient’s veins. Prescription of the treatment products depends on the type of care given (prophylactic/episodic).

Theoretically, Gene therapy is more efficacious than Replacement therapy, as it can help form clotting factors in a patient, which can further help restore normal blood clotting. Therefore, Gene therapy for Hemophilia is favoured as a revolutionary and an advanced treatment option. However, even though gene therapy looks promising, it also has a few drawbacks.

Drawbacks:

• The method of treatment involved uses viruses that can potentially trigger an immune reaction or response in a patient. Thus, any antibodies produced can kill the viruses, and stop the treatment from happening at all.
• Another drawback is the acute risk factor that a patient’s body might have to sustain when being operated on with gene therapy treatment for Hemophilia.
• Inconsistent results are another drawback when considering gene therapy for Hemophilia treatment. While studies conducted on animals have shown expected results, the same hasn’t been found in human trials yet.
• Deciding between gene therapy and other options can be extremely difficult since the complications associated with gene therapy are high.

Benefits:

• Hemophilia is a monogenic disease(a single genetic defect disorder), which makes it an ideal prospect for gene therapy mechanisms, producing highly effective results.
• Another reason that makes gene therapy likely successful is that even small successes from the procedure can significantly improve the patient’s quality of life.
• How Hemophilia occurs in terms of a genetic disorder is another considerable advantage in gene therapy for Hemophilia. Compared to other genetic disorders involving numerous genetic defects, Hemophilia proves to be an easier target for gene therapy.

Resources:Gene Therapy For Hemophilia, By Delveinsight.