Scientists designed an easier gene transfer technique through Lentiviral Vector, for enhancing the production of Foetal Haemoglobin.
Red blood cells, are most crucial in case of any mammalian species as it helps in the transfers of oxygen throughout the body. Produced from the process of Hematopoiesis, red blood cells also known as Erythrocytes, have a characteristics shape and size directly relatable to their function of oxygen transport.
Sickle Cell Anemia or Sickle Cell Disease (SCD), is common inheritable genetic disorder of the Erythrocytes. In this disease an Erythrocytes changes its shape to convert itself in to a structure that resembles a “sickle” used in framing, from roundish, hollow structure. This change in the conformation cause the “sickled” red blood cells to undergo early death via necrotic pathway. Thereby, a patient experience shortage in the blood count, which gives rise to series of other problems in the body like stroke, chest pain, organ damage etc.
Owing to its genetic occurrence, that is mutation in the Hemoglobin- Beta gene (Hb-β) present on the chromosome 11, Genetic therapy, to cure this disease can be very potent option. SCD, is very common in African population, the reason being the lack of proper nutrition among them from birth. In some cases scientist found the disease to be highly relatable with that of β-Thalassemia, in which the erythrocyte instead of being sickled in shape, is becoming smaller, but causing the same effect of impaired oxygen transport.
The main moto of the gene therapy regarding this aspect encircles the production of a healthy Erythrocytes, in proper shape and size, so that they can transfer, oxygen throughout the body, in proper amount. The genetic therapy strategy, boosts the production of haemoglobin during foetal condition by increasing the concentration of the gamma-Globin protein. Foetal Haemoglobin has a better efficiency in transport function. Stimulating the production of Foetal Haemoglobin can also reduce the chances of the occurrence of SCD.
Previous method of Hematopoietic Stem Cell stimulation is a robust as well as complex Lentiviral transfer technique, for curing complication related to SCD. Thus, it can also say that in “not-too-distant future”, a better therapeutic model will arrive, which will cure SCD, and other Hematopoietic disorders.