Is gene therapy part of the treatment and what current research before this disease is being done in the area of genetic engineering?
Sickle cell disease is one of the genetic disorders. On the other hand, gene therapy is a treatment technique that uses genes to treat such genetic diseases. This is one of the facts that approves gene therapy as part of the treatment of sickle disease. Research indicates that sickle disorder affects hemoglobin, the molecule responsible for circulating oxygen throughout the body (Ribeil et. al, 2017). Although bone marrow transplant is the only proved treatment approach, current research indicates that gene therapy can be employed to treat sickle cell disease. Unlike the identified treatment approach, gene therapy involves the use of genetic materials harvested from the patient’s bone marrow to compensate for the sick genes in the body system. The gene inserted into a cell is genetically engineered into the body using viruses that infect the cells hence transmitting the required genes.
Delegate your assignment to our experts and they will do the rest.
Before the approach is employed for the treatment of the identified disorder, research affirms various aspects of the treatment that would influence its application. Gene therapy does not require donor participation since genes are harvested from the patient’s bone marrow. Gene therapy eliminates the concern of making the patient vulnerable to infections be donor’s cells. In addition, the genes are carefully selected to correct the effect of a mutated gene that is causing the sickle disorder. Therefore, the research that is being done concerning the use of gene therapy for treatment of sickle disorder involves determining the effectiveness of introducing new gene in the immune system to help fight sickle disease (Ribeil et. al, 2017). Furthermore, research is being carried out to shorten the intensive procedure that is used to make the treatment approach successful. Current research involving clinical trials is also being carried out to determine whether gene therapy is effective for curing sickle cell disorder. In the future, gene therapy will be considered for the treatment of sickle cell disorder following its numerous benefits as a treatment technique.
References
Ribeil, J. A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., ... & Bartolucci, P. (2017). Gene therapy in a patient with sickle cell disease. New England Journal of Medicine , 376 (9), 848-855.
