Introduction
Gene therapy is a technological approach employed to combat hereditary illnesses and to replace defective genes. This procedure entails the elimination of the defective gene or modification of the gene to allow it to do its work normally and then replacing the gene so that it improves the underlying ailment. In the context of sickle cell anemia, gene therapy aims for a functional form of the β-globin gene to substitute the defective product of the mutation (Leonard & Tisdale, 2021). This paper aims to provide background information about the use of gene therapy in eradicating sickle cell anemia and the steps that have been taken to make gene therapy possible. The paper will also discuss the societal impact of current and potentially future strategies.
Background of the topic
Sickle Cell anemia is a hereditary red blood cell disorder that arises due to a genetic mutation that leads to an abnormal protein structure. Specifically, it is caused by a mutation in the hemoglobin beta genes. According to Orkin & Bauer (2019), the mutation alters the red blood cell hemoglobin structure to take on a shape more conducive to fracture and decreases the red blood cell membrane’s capability to transport oxygen throughout the whole body. These cells also become easily damaged and clump together, blocking small blood vessels. This disorder is an inherited trait with human carriers presenting two genes for the mutated hemoglobin beta, resulting in an imbalance of charge between the two molecules resulting in bulging on the surface of the red blood cells. This causes the sickle shape, creating health problems requiring consistent medical attention. With some treatments providing limited improvement, most people living with it stay stuck, suffering pain and organ damage caused by these acquired deformities. Moreover, People affected by SCD generally exist in extreme pain, have shortened life expectancy, as well as have weakened bones, and have a higher probability of having strokes.
Steps that have been done to enhance the possibility of the use of gene therapy in eradicating sickle cell anemia
The major research of gene therapy for SCA has been ongoing for over two decades, and recently, a significant breakthrough suggests this technology effectively be used against the faulty gene which has been accountable for causing it. There have been several experimental trials that are focused on using gene therapy, principally on SCA-affected patients. A well-known attempt at delivering what is known as the “Bolton/Graham model” was performed in 2015, where a patient was subjected to gene therapy insertion as soon as they were born (Leonard & Tisdale, 2021). Following an ordinary form of birth, a lentiviral-based vector had been infected with a prodromal beta-globin gene in their body. Additional modifications to the treated vector caused attenuation from the host immune reaction from incoming copies and are currently being applied in more practices.
Another more recent technique for introducing strains of targeted genetically altered red blood cells has been implanted using cell factor VIII and taken up by T-lymphocytes out of transfemoral units using a vein cuffing procedure. At first, results from this technique were not positive in most situations, but further investigations have acted to modify this function. With additional systemic conditioning with Hydroxyurea pharmacological cocktail and protection substances, their aptitude for changing and providing viable remedies has increased. Additionally, long-term laboratory and animal studies have provided evidence of actual expression with typical modifications, but more research is still needed.
Societal impact of current and potentially future strategies.
The pandemic caused by SCA still remains largely unrivaled, and treatment remains exclusive for most affected individuals. Mass consideration and further prospective immunomodulatory treatments may benefit from further public-funded research opportunities and advancements in diagnostic strategies; nonetheless, government investment for the technology involved in the inclusive genetic study for SCA still needs to be addressed, particularly in impoverished countries like India and Subactivities states.
As various genetic inquiries and therapies focusing on pathogenetic traits develop, we can have partial cures or a total recovery rate, especially for pediatric people affected by inherited disorders. We can expect proper dispensations of cell-engineered attributes and alleviate clinical issues anemically related. Also, this could lead us to more personalized treatments involving reinforced genetic design (Sinha et al., 2021). This also may extend us closer to having regulatory overlying, which may guard critical factors like equitable proof of benefit versus harm risk allocation or informed consent before implementation concerning especially reckless methodology like germ line therapy or mosaicism inside engineered genes processing chain, towards safe accommodating socioeconomic judicial contexts.
Conclusion
In conclusion, gene therapy has the potential to cure sickle cell anemia by replacing defective genes with functionally associated ones or managing factors that artificially affect hemoglobin levels as components furthered to achieve satisfactory levels, such as radiation-mediated marrow prior to crystalloid transduction. Though promising, much research still needs to be done before we can say for sure that this technology is reliable for therapeutic use, but when it comes to supplying needed care for those affected by SCA, it is hopeful that these novel platforms can become accessible.
References
Leonard, A., & Tisdale, J. F. (2021). A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease. Molecular Therapy, 29(4), 1355-1356.
Sinha, C. B., Bakshi, N., Ross, D., Loewenstein, G., & Krishnamurti, L. (2021). Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease. Pediatric blood & cancer, 68(1), e28749. https://doi.org/10.1002/pbc.28749
Orkin, S. H., & Bauer, D. E. (2019). Emerging genetic therapy for sickle cell disease. Annual review of medicine, 70, 257-271.
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