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CRISPR Gene Editing and Its Potential in Curing Genetic Diseases
Subject area: Science,Engineering and Technology · Area of research: B.E.T Sadathunnisa Degree College
Abstract
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene editing has emerged as a revolutionary tool in molecular biology, offering unprecedented precision and efficiency in modifying genetic material. By utilizing the Cas9 protein guided by a custom-designed RNA sequence, CRISPR enables targeted cutting and alteration of DNA at specific locations. This technology holds immense potential for curing a wide range of genetic diseases, including sickle cell anemia, cystic fibrosis, muscular dystrophy, and Huntington?s disease, by correcting the underlying mutations responsible for these disorders. Recent clinical trials have demonstrated promising results, particularly in the treatment of blood-related conditions such as beta- thalassemia and sickle cell disease. However, challenges such as off-target effects, ethical concerns regarding germline editing, and equitable access to CRISPR-based therapies remain significant barriers. With ongoing research and improvements in precision techniques like prime editing and base editing, CRISPR could pave the way for a new era of personalized medicine, offering permanent cures for previously untreatable inherited conditions. This paper explores the mechanism of CRISPR, its applications in treating genetic diseases, and the ethical considerations surrounding its clinical use.
How to cite this paper
@article{1711049,
author = {Sunar Muskan , Zunaira K. A},
title = {CRISPR Gene Editing and Its Potential in Curing Genetic Diseases},
journal = {Iconic Research And Engineering Journals},
year = {2025},
volume = {9},
number = {4},
pages = {266-269},
issn = {2456-8880},
url = {https://www.irejournals.com/formatedpaper/1711049.pdf},
abstract = {CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene editing has emerged as a revolutionary tool in molecular biology, offering unprecedented precision and efficiency in modifying genetic material. By utilizing the Cas9 protein guided by a custom-designed RNA sequence, CRISPR enables targeted cutting and alteration of DNA at specific locations. This technology holds immense potential for curing a wide range of genetic diseases, including sickle cell anemia, cystic fibrosis, muscular dystrophy, and Huntington?s disease, by correcting the underlying mutations responsible for these disorders. Recent clinical trials have demonstrated promising results, particularly in the treatment of blood-related conditions such as beta- thalassemia and sickle cell disease. However, challenges such as off-target effects, ethical concerns regarding germline editing, and equitable access to CRISPR-based therapies remain significant barriers. With ongoing research and improvements in precision techniques like prime editing and base editing, CRISPR could pave the way for a new era of personalized medicine, offering permanent cures for previously untreatable inherited conditions. This paper explores the mechanism of CRISPR, its applications in treating genetic diseases, and the ethical considerations surrounding its clinical use.},
month = {October},
}