ARTICLE TYPE : RESEARCH ARTICLE
Published on : 19 Sep 2025, Volume - 1
Journal Title : WebLog Journal of Hematology | WebLog J Hematol
Source URL: 
https://weblogoa.com/articles/wjh.2025.i1902
Permanent Identifier (DOI) : 
https://doi.org/10.5281/zenodo.17442104
Therapeutic Potential of CRISPR-Cas9 in Treating Inherited Hematological Disorders
2Assistant Professor, Department of Pathology, Dow University of Health Sciences (DUHS), Karachi, Pakistan
Abstract
Inherited hematological disorders, to a degree, weapon cell affliction, thalassemia, and hemophilia, have long been a challenge in clinical care, accompanying restricted therapeutic alternatives. These disorders are caused by ancestral mutations that hinder the function of blood cells, leading to lifelong obstacles. Traditional situations, containing blood transfusions and bone marrow transplants, offer only temporary relaxation and are frequently associated with significant risks. Recent progress in DNA editing sciences, specifically CRISPR-Cas9, offers new hope for the situation of these ancestral ailments. This paper explores the potential of CRISPR-Cas9 as a healing form for inherited hematological disorders. By targeting a specific point or directly at a goal and correcting genetic mutations at the DNA level, CRISPR-Cas9 can conceivably cure these disorders by enabling the result of athletic ancestry cells. Early dispassionate tests have shown hopeful results in doctoring environments, such as curing container disease and testing-thalassemia, where DNA editing methods have been used to modify hematopoietic stem cells and replace common blood cell function. However, challenges to a degree, wide effects, transfer adeptness, and long-term security are expected to be addressed. This paper reviews the current state of CRISPR-Cas9-based analyses for inherited hematological disorders, evaluates the moral concerns, and reviews future directions for research and dispassionate requests. The therapeutic potential of CRISPR-Cas9 shows a pioneering shift in the situation of genetic ailments, contributing to the possibility of permanent cures for patients suffering from inherited blood disorders.
Keywords: CRISPR-Cas9; Gene Editing; Inherited Hematological Disorders; Sickle Cell Disease; Thalassemia; Hemophilia; Gene Therapy; Genetic Mutations; Stem Cells; Clinical Trials; Genetic Med
Citation
Haider R, Abbas H. Therapeutic Potential of CRISPR-Cas9 in Treating Inherited Hematological Disorders. WebLog J Hematol. wjh.2025.i1902. https://doi.org/10.5281/zenodo.17442104