CRISPR Cas-9: Revolutionary Genome Editing Poses Ethical Challenges

CRISPR Cas-9: Revolutionary Genome Editing Poses Ethical Challenges
Imagine you are living in a world where we can edit genes as easily as correcting a word in a book of five volumes, a typo in a text message, or fixing a broken zipper – a world where genetic disorders are no longer the things to be worried about and we have the tools and power to reshape them. This sounds like science fiction, but thanks to CRISPR, a revolutionary gene editing tool, we are now able to do just that in the real world. The mechanism is a natural defense system of bacteria and has tremendous potential to unlock the realm of genetic engineering. The ability to shape our genetic destiny now lies in your fingerprints.

In 2012, four scientists - George Church, Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang revealed that this bacterial immune system could be manipulated as a powerful tool for editing genes. They discovered a protein called Cas9, which is an integral part of the CRISPR system to modify desired regions of the DNA in different organisms. And because of this remarkable development in genome editing the year 2015, was named the “CRISPR Breakthrough of the Year” by the journal Science. More interestingly, for the first time in history, two women Jennifer Doudna and Emmanuelle Charpentier were awarded the Nobel Prize for their contributions to the CRISPR Cas-9 system by identifying “Genetic incisors,” an analogy for the cutting scissors that are used to tailor cloth to size.

Who owns CRISPR-Cas9? The CRISPR-Cas9 patent dispute is also worth mentioning. A study revealed that there are more than ten thousand patents on CRISPR-related technology. Most famously, two groups have claimed the right to use this gene editing tool for eukaryotic cells: the University of California, Berkeley, University of Vienna, and Dr. Charpentier on one side, and Harvard-MIT-Broad Institute on the other hand. This dispute has resulted in a complex and lengthy process at the US Patent Office and the court. Initially, The Broad Institute succeeded in getting a patent in the US, while the European patent was won by the University of California. This whole episode of complex situations caused a serious issue for biotech companies using CRISPR-Cas9 technology.

The real-time applications of CRISPR are gene therapy, medicine development, and genome manipulation of human somatic cells. Initially, vectors were being used for gene therapy. Now, genetic editing tools like CRISPR brought new possibilities. In 1916, the first clinical trial of CRISPR began in China, followed by the US in 2019 to treat sickle cell anemia. However, this emerging technology is not cost-effective. The well-known therapy in the market is Zolgensma, used to treat muscular atrophy, with an estimated cost of $1.5 million. In 2017, Sangamo Therapeutics accomplished a significant feat by successfully treating a patient with Hunter Syndrome using Zinc Finger Nuclease (ZFNs). Another remarkable milestone was reached in 2020 by Editas Medicine, which injected a drug named EDIT-101 into the retina of a patient with inherited blindness.

The technology is still in its infancy. Randomized clinical trials are underway to explore the vast potential of CRISPR in treating various diseases, including Alzheimer's, cancer, baldness, and high cholesterol. Victoria Gray, a 37-year-old survivor of the disease is a famous success story. Her painful story about the complex and severe symptoms that had disrupted her childhood was a challenge for clinical practitioners. Her children were worried she might die. Billions of bone marrow cells had been trained and modified using CRISPR and reinfused into her body. Thanks to CRISPR Cas-9, smiles returned to her face.

A gene therapy procedure called CAR T, which modifies the patient’s immune system to specifically target the cancer cell, also achieved insightful results. With a remission rate of up to 85% in treating lymphoma and acute lymphoblastic, the treatment has shown significant outcomes. Additionally, better management has helped mitigate severe side effects. Furthermore, there is a promising outlook for utilizing CRISPR to combat infectious diseases such as human papillomavirus and HIV shortly.

CRISPR-Cas9 has tremendous potential for improving agricultural outputs. Increasing crop yield has become inevitable vis-à-vis the burgeoning world population, threatening food security. An integrated approach is needed to address challenges faced by plants in tropical regions of Asia.

The ethical aspects and safety of gene therapy using CRISPR-Cas9 demand thorough discourse. The first concerns appeared shortly after publications on human cells. Scientists warned that heritable changes in human embryos, without considering the social, environmental, and ethical consequences, could be challenging. A debate in Nature also emphasized the negative consequences and ethical considerations of the CRISPR-Cas9 system. Historical medical experimentation scandals, like the Tuskegee syphilis study, led to the development of international bioethics guidelines. In 2018, a Chinese scientist Jiankui, announced the creation of genetically modified babies using CRISPR-Cas9, making the guidelines more relevant. Jiankui ended up in prison and was released last year. Today, strict laws have been enacted in China for heritable genome editing.

The scientific community was shocked and expressed serious concerns associated with intentional mutations that can lead to severe ethical consequences. Another Russian scientist also expressed his intention to create genetically modified babies, but emphasized the need for strict regulatory and ethical approval. Of late, Würzburg, Germany hosted the CRISPR 2023 Conference from 27 June to 1 July. The conference covered various aspects of CRISPR biology: genetics, structural biology, biochemistry, ecology, evolution, and applications.

The legal framework associated with genome editing varies among different countries. Some countries allow non-reproductive genome modifications, while others prohibit such experiments. Manipulation in the reproductive genome is a serious concern and must be addressed. Artificial intelligence, web3, quantum computing, virtual realities, and CRISPR in biology are emerging fields that have the potential to revolutionize the scientific landscape. It is imperative for advanced countries to proactively facilitate the equitable transfer of knowledge globally, recognizing the need of the time.

The author is a PhD candidate at the Atta-ur-Rahman School of Applied Biosciences, National University of Sciences and Technology (NUST) in Islamabad