Volume : 13, Issue : 06, June – 2026
Title:
EPIDEMIOLOGICAL TRENDS AND COMPARATIVE CLINICAL EFFICACY OF CRISPR-CAS9 VERSUS HEMATOPOIETIC STEM CELL TRANSPLANTATION IN TRANSFUSION-DEPENDENT \BETA-THALASSEMIA: A SYSTEMATIC REVIEW
Authors :
Musarrath Mubeen*, D.deppika, k.savithri, J.lavanya, B.balu,,T.Mangilal
Abstract :
Beta thalassemia is a hereditary blood disorder characterized by reduced or absent synthesis of β-globin chains, leading to ineffective erythropoiesis and chronic haemolytic anaemia. This systematic review aimed to evaluate the prevalence, clinical manifestations, associated complications, and current treatment approaches of beta thalassemia. Data from multiple published studies involving a large patient population were analysed. The findings indicate a higher prevalence in regions such as South Asia, particularly India, due to genetic factors and consanguineous marriages. Clinical features commonly include severe anaemia, growth retardation, splenomegaly, and bone deformities.
Complications such as iron overload, cardiac disorders, liver dysfunction, endocrine abnormalities (including diabetes mellitus and hypothyroidism), and increased susceptibility to infections were frequently reported. Conventional management strategies include regular blood transfusions and iron chelation therapy to manage iron overload. Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment, though its availability is limited due to donor compatibility and cost constraints. Emerging therapies such as gene therapy and CRISPR-Cas9 gene editing show promising potential but are still under development and not widely accessible, especially in developing countries like India. The study highlights the importance of early diagnosis, genetic counselling, and preventive screening programs to reduce disease burden. A multidisciplinary approach involving haematological, genetic, and supportive care is essential to improve patient outcomes and quality of life.
Keywords: Beta thalassemia, Haemoglobin disorder, Genetic mutation, Anaemia, Iron overload, Blood transfusion, Iron chelation therapy, HSCT, Gene therapy, CRISPR-Cas9.
Cite This Article:
Please cite this article in press Musarrath Mubeen et al., Epidemiological Trends And Comparative Clinical Efficacy Of Crispr-Cas9 Versus Hematopoietic Stem Cell Transplantation In Transfusion-Dependent \Beta-Thalassemia: A Systematic Review.., Indo Am. J. P. Sci, 2026; 13(06).
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