Vol. 9, Issue 11, Part D (2025)
Advances in therapeutic approaches: Hydroxyurea, stem cell transplantation, and gene therapy
Pragati Gupta, Meenoo Kumbhar and Shani Raj
The mortality and morbidity rates associated with sickle cell disease (SCD) are significant all over the globe; however, they are particularly high in regions where the illness is prevalent, such as India, the Middle East, and sub-Saharan Africa. Over the course of the last three decades, advancements in therapeutic intervention have brought about a revolution in the treatment of sickness. This change in emphasis from symptomatic therapy to curative and customized treatments has occurred. The Food and Drug Administration (FDA) first authorized hydroxyurea as a medication to treat sickle cell disease (SCD). Acute chest syndrome, vaso-occlusive crises, and transfusion requirements are all alleviated. However, there are a number of drawbacks to this therapy, including issues with accessibility, long-term safety, and the variability in patient response. Stem cell transplantation, and in particular allogeneic hematopoietic stem cell transplantation (HSCT), presents a promising therapeutic alternative; yet, its use is limited by a multitude of circumstances. Donor availability, risk of graft-versus-host disease, and financial constraints are all relevant considerations. The use of viral vector systems, CRISPR-Cas9, and molecular genetics, among other recent developments in gene therapy, have shown promising clinical research prospects as a method to correct or compensate for the underlying mutation in the β-globin gene. The results of meta-analyses, PRISMA-based systematic reviews, and visual comparisons are all a part of this study. A combination of recent findings in genetics, treatment effectiveness, and epidemiological data forms the basis of the research. We can show how treatments work, how genes work, and what the future holds by using visualization tools like histograms, forest plots, and route diagrams. Our main goal is to promote sickle cell disease (SCD) precision medicine by connecting molecular genetics with clinical applications. In the article's latter sections, the essayists highlight the need of ensuring that diverse populations have fair access to novel treatments, as well as the current constraints and financial obstacles.
Pages: 286-293 | 69 Views 34 Downloads

