The rapid development of gene editing technology has brought unprecedented hope and challenges to humanity. This technology can directly alter human genes, thereby preventing or treating a series of genetic diseases. However, gene editing technology has also raised many ethical issues, which could lead to permanent changes in the human genome and potential harm. This article will explore the application fields, impact, and challenges of gene editing technology, in order to provide readers with a comprehensive understanding of this topic.
1. Definition and Principle of Gene Editing Technology
Gene editing technology refers to the process of modifying, deleting, replacing, or inserting human genes using specific molecular tools. The basic principle of this technology is to use the CRISPR-Cas9 system or other similar nucleases to cut the DNA chain and introduce specific changes at the cut site. This change can be to repair the DNA chain or insert a new DNA sequence. In this way, gene editing can change specific parts of the human genome to achieve the purpose of treating or preventing diseases.
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2. Application Fields and Current Status of Gene Editing Technology
Gene editing technology has been widely applied in the medical field. For example, gene editing can cure some stubborn genetic diseases, such as cystic fibrosis, hemophilia, and thalassemia, etc. In addition, gene editing technology can also be used to improve human physical fitness, such as increasing height, enhancing muscle strength, and improving vision, etc. At present, gene editing technology has entered the clinical trial stage and has achieved some exciting results.
3. Impact and Challenges of Gene Editing Technology on the Future of Humanity
The rapid development of gene editing technology brings great hope and challenges to humanity. On the one hand, gene editing technology can help humanity cure some untreatable genetic diseases, improve human physical fitness, and extend human life expectancy. On the other hand, the misuse of gene editing technology may also lead to a series of serious ethical issues, such as changing the course of human evolution, causing genetic discrimination, and threatening biodiversity. Therefore, we need to formulate relevant laws, regulations, and ethical guidelines to regulate the application and development of gene editing technology, to ensure that the application of this technology is carried out within a safe and legal range.
According to public information, gene editing technology has been applied to the fields of cancer, genetic diseases, and immune diseases, among which several drugs have been approved for marketing, such as Novartis's Zolgensma, Bayer's Stivarga, and Verily's Ver aneur, etc.
Zolgensma (AVXS-101) is a gene therapy drug developed by Novartis, a pharmaceutical company in the United States, for the treatment of spinal muscular atrophy (SMA). Zolgensma uses the AAV9 vector to mediate the replacement of the SMN1 gene, rather than gene editing technologies such as CRISPR. On May 24, 2021, Zolgensma was approved by the FDA for marketing and is the first gene therapy drug for SMA.The gene editing therapy CTX (Kymriah) is a CAR-T cell therapy, co-developed by Novartis and UPenn, for the treatment of acute lymphoblastic leukemia. In August 2017, Kymriah was approved by the FDA for marketing, becoming the first approved CAR-T cell product, but not the first gene-edited CAR-T cell product.
Tecentriq is a PD-L1 inhibitor used for the treatment of malignant melanoma, non-small cell lung cancer, and urothelial cancer. Tecentriq is not a gene-edited drug. In 2018, Tecentriq was approved for the treatment of advanced urothelial cancer and non-small cell lung cancer.
Kite Pharma's CAR-T cell therapy, Yescarta, is used for the treatment of diffuse large B-cell lymphoma. In October 2017, Yescarta was approved by the FDA for marketing, and it is also not the first CAR-T cell product to be marketed.
In China, although gene editing technology has made some progress, such as the approval of the Cartacef series of gene editors around 2015, there are currently no gene-edited drugs approved for marketing. However, some hospitals in China have introduced gene editing technology and have carried out some related projects in clinical trials.
Here is some information on Chinese hospitals that have introduced gene editing technology:
Peking Union Medical College Hospital: The hospital established a gene editing technology research center in 2016 and is currently conducting multiple gene editing clinical trials, including treatment plans for cancers such as lung cancer, liver cancer, and leukemia, as well as gene editing treatment plans for genetic diseases.
Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine: The hospital established a gene editing technology research center in 2018 and is currently conducting multiple gene editing clinical trials, including treatment plans for genetic eye diseases, hemophilia, and immune diseases.
Sun Yat-sen University Cancer Center: The hospital introduced gene editing technology in 2017 and is currently conducting clinical trials for cancers such as liver cancer, lung cancer, and breast cancer.
Xijing Hospital of Air Force Medical University: The hospital introduced gene editing technology in 2018 and is currently conducting clinical trials for diseases such as genetic eye diseases, hemophilia, and immune diseases.It should be noted that gene editing technology is still in its early stages in clinical application, and the safety and efficacy of treatment plans need further verification. In addition, the cost of gene editing technology is relatively high, and currently only a small number of patients can afford this high-end treatment.
4. Future Prospects
The rapid development of gene editing technology has brought endless possibilities for humanity. In the future, we can use gene editing technology to cure more genetic diseases, improve human physical fitness, and extend human life expectancy. At the same time, we also need to strengthen the formulation and implementation of relevant laws, regulations, and ethical guidelines to ensure that the application of gene editing technology is carried out within a safe and legal framework. Moreover, we need to enhance public communication and science popularization work to let more people understand the potential impacts and challenges of gene editing technology, in order to promote the sustainable development and application of this technology.
In summary, gene editing technology has brought endless possibilities for humanity, but it also requires us to think carefully and deal with potential ethical issues and social challenges. Only with the joint promotion of science, ethics, and society can gene editing technology better serve humanity and create a better future.