International Journal of Molecular Medical Science, 2024, Vol.14, No.1, 8-15 http://medscipublisher.com/index.php/ijmms 10 Figure 1 Application of CRISPR/Cas9 Despite the numerous advantages of CRISPR-Cas9 technology, it also faces several challenges. For instance, unintended cutting or editing events may occur, leading to unexpected genetic alterations. Additionally, the application of CRISPR-Cas9 technology in specific tissues or cell types poses challenges. However, with ongoing technological improvements, it is foreseeable that CRISPR-Cas9 will become a powerful and widely utilized gene editing tool. In summary, CRISPR-Cas9 offers higher precision, flexibility, cost-effectiveness, and shorter working times compared to traditional gene editing techniques. 2 The History and Development of Gene Therapy 2.1 Overview of traditional gene therapy approaches Traditional gene therapy methods refer to non-CRISPR-related techniques used to repair or regulate human genes. These approaches hold potential in the treatment of certain diseases, particularly monogenic disorders. However, they also face challenges such as low delivery efficiency, cell specificity, immune responses, and long-term stability. Traditional gene therapy methods include gene replacement, gene correction, and gene suppression, among others. Gene replacement involves introducing a normal gene into a patient's body to replace a defective gene. This can be achieved by delivering the normal gene into the patient's cells using vectors, such as viral vectors or plasmid DNA. Once the normal gene is introduced and expressed, it can correct the genetic defect and restore normal function. Gene correction refers to the correction or modification of erroneous gene sequences to rectify genetic defects. This can be achieved by using DNA modifying enzymes such as zinc finger nucleases and transcription active factors (Belete, 2021). Gene suppression involves inhibiting the expression of a defective gene through the use of interfering RNA (siRNA) or antisense RNA. Interfering RNA is a short RNA molecule that can interact with specific gene sequences in patient cells, selectively reducing or inhibiting the expression of the targeted gene. Antisense RNA is an RNA molecule that prevents its translation into a protein by interacting with the mRNA molecule of the target gene. 2.2 Progress and limitations of gene therapy Gene therapy is an emerging therapeutic approach that has made significant strides over the past few decades. In terms of clinical applications, gene therapy has successfully been used for some genetic diseases, such as severe combined immunodeficiency and familial hypercholesterolemia (Godbout and Tremblay, 2023). Furthermore, the genetic engineering of patient T cells to express chimeric antigen receptors (CARs) has been successfully applied
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