AI Article Synopsis
- CRISPR/Cas9 is a powerful gene editing tool that leverages a bacterial immune system and has potential applications in preventing and treating a wide range of diseases, including cancers and genetic disorders.
- Its advantages over other gene editing methods include higher specificity and efficiency, making it a focus of many clinical trials for various illnesses.
- The text also addresses challenges like off-target effects and discusses methods for delivering CRISPR/Cas9 into cells, including both viral and non-viral vectors.
Article Abstract
CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced short palindromic repeats) as a bacterial immune system and plays as a gene editing tool. Due to the higher specificity and efficiency of CRISPR/Cas9 compared to other editing approaches, it has been broadly investigated to treat numerous hereditary and acquired illnesses, including cancers, hemolytic diseases, immunodeficiency disorders, cardiovascular diseases, visual maladies, neurodegenerative conditions, and a few X-linked disorders. CRISPR/Cas9 system has been used to treat cancers through a variety of approaches, with stable gene editing techniques. Here, the applications and clinical trials of CRISPR/Cas9 in various illnesses are described. Due to its high precision and efficiency, CRISPR/Cas9 strategies may treat gene-related illnesses by deleting, inserting, modifying, or blocking the expression of specific genes. The most challenging barrier to the use of CRISPR/Cas9 like off-target effects will be discussed. The use of transfection vehicles for CRISPR/Cas9, including viral vectors (such as an Adeno-associated virus ()), and the development of non-viral vectors is also considered.
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|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10425811 | PMC |
| http://dx.doi.org/10.1016/j.gendis.2023.02.027 | DOI Listing |
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