Recent Therapeutic Gene Editing Applications to Genetic Disorders

Recent Therapeutic Gene Editing Applications to Genetic Disorders

30 April 2024 | Eric Deneault
The review discusses the recent advancements in therapeutic gene editing, particularly focusing on the emergence and application of CRISPR-based technologies. CRISPR-Cas9 nuclease, base editing, and prime editing have demonstrated remarkable precision and efficacy in targeted ex vivo and in vivo genomic modifications. Enhanced delivery systems, such as viral vectors and nanoparticles, have improved the efficiency and safety of therapeutic gene editing. The exploration of CRISPR-Cas systems beyond Cas9, including Cas12 and Cas13 variants, has expanded the repertoire of gene editing tools. Prime editing, in particular, represents a significant advancement due to its versatility and minimal off-target effects. These innovations have paved the way for therapeutic gene editing in a wide range of genetic disorders, from monogenic diseases to complex polygenic conditions. However, challenges such as off-target effects and ethical considerations remain, necessitating continued research to refine safety profiles and ethical frameworks. The review highlights the latest innovative studies in preclinical and clinical trials, emphasizing breakthrough technologies and their applications in precision medicine.The review discusses the recent advancements in therapeutic gene editing, particularly focusing on the emergence and application of CRISPR-based technologies. CRISPR-Cas9 nuclease, base editing, and prime editing have demonstrated remarkable precision and efficacy in targeted ex vivo and in vivo genomic modifications. Enhanced delivery systems, such as viral vectors and nanoparticles, have improved the efficiency and safety of therapeutic gene editing. The exploration of CRISPR-Cas systems beyond Cas9, including Cas12 and Cas13 variants, has expanded the repertoire of gene editing tools. Prime editing, in particular, represents a significant advancement due to its versatility and minimal off-target effects. These innovations have paved the way for therapeutic gene editing in a wide range of genetic disorders, from monogenic diseases to complex polygenic conditions. However, challenges such as off-target effects and ethical considerations remain, necessitating continued research to refine safety profiles and ethical frameworks. The review highlights the latest innovative studies in preclinical and clinical trials, emphasizing breakthrough technologies and their applications in precision medicine.
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Understanding Recent Therapeutic Gene Editing Applications to Genetic Disorders