Recent Therapeutic Gene Editing Applications to Genetic Disorders

Recent Therapeutic Gene Editing Applications to Genetic Disorders

2024 | Eric Deneault
Recent advances in therapeutic gene editing have transformed the treatment of genetic disorders. This review highlights the progress in CRISPR-based technologies, including CRISPR-Cas9, base editing, and prime editing, which enable precise and targeted modifications of the human genome. These tools have been enhanced by improved delivery systems, such as viral vectors and nanoparticles, increasing their clinical translatability. Variants like Cas12 and Cas13 have expanded the range of gene editing applications, while prime editing offers high versatility and minimal off-target effects. These innovations have enabled therapeutic interventions for previously incurable genetic disorders, from monogenic to complex polygenic conditions. However, challenges such as off-target effects and ethical concerns remain, requiring further research to refine safety and ethical frameworks. The review also discusses the molecular mechanisms of CRISPR, the development of next-generation CRISPR-Cas9, and the application of gene editing in various diseases, including blood disorders, neurological conditions, and infectious diseases. Delivery systems, such as viral vectors and nanoparticles, are crucial for efficient and safe gene editing. The review highlights successful clinical trials and preclinical studies in treating conditions like sickle cell disease, beta-thalassemia, severe combined immunodeficiency, chronic granulomatous disease, hemophilia, and neurological disorders such as ALS, Alzheimer's, Huntington's, and Niemann-Pick disease. These studies demonstrate the potential of gene editing to correct genetic mutations, improve disease outcomes, and offer long-term therapeutic solutions. Despite challenges, the field continues to advance, with ongoing efforts to enhance precision, reduce off-target effects, and expand applications in medicine.Recent advances in therapeutic gene editing have transformed the treatment of genetic disorders. This review highlights the progress in CRISPR-based technologies, including CRISPR-Cas9, base editing, and prime editing, which enable precise and targeted modifications of the human genome. These tools have been enhanced by improved delivery systems, such as viral vectors and nanoparticles, increasing their clinical translatability. Variants like Cas12 and Cas13 have expanded the range of gene editing applications, while prime editing offers high versatility and minimal off-target effects. These innovations have enabled therapeutic interventions for previously incurable genetic disorders, from monogenic to complex polygenic conditions. However, challenges such as off-target effects and ethical concerns remain, requiring further research to refine safety and ethical frameworks. The review also discusses the molecular mechanisms of CRISPR, the development of next-generation CRISPR-Cas9, and the application of gene editing in various diseases, including blood disorders, neurological conditions, and infectious diseases. Delivery systems, such as viral vectors and nanoparticles, are crucial for efficient and safe gene editing. The review highlights successful clinical trials and preclinical studies in treating conditions like sickle cell disease, beta-thalassemia, severe combined immunodeficiency, chronic granulomatous disease, hemophilia, and neurological disorders such as ALS, Alzheimer's, Huntington's, and Niemann-Pick disease. These studies demonstrate the potential of gene editing to correct genetic mutations, improve disease outcomes, and offer long-term therapeutic solutions. Despite challenges, the field continues to advance, with ongoing efforts to enhance precision, reduce off-target effects, and expand applications in medicine.
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