To minimize the risk, the team tested several other approaches, such as modified versions of CRISPR components. Or use protective elements that reduce the likelihood that the genetic scissors will cut the chromosome in several places at the same time. Unfortunately, none of these measures could completely prevent the unwanted side effects.
“This study shows the promising and challenging aspects of CRISPR-based therapies,” says co-author Martin Jinek, professor at the UZH Institute of Biochemistry. It provides valuable clues to the development of gene editing therapies for chronic granulomatosis and other hereditary diseases. “But further technological advances are needed to make the method safer and more effective in the future.”
literature
Federica Raimondi et al. Gene editing at NCF1 loci is associated with homologous recombination and chromosomal rearrangements. Communication Biology. 9 October 2024. DOI:
Interview with Martin Jinek on the Future of CRISPR-Based Therapies
Editor: Thank you for joining us today, Professor Jinek. Your recent study sheds light on the complexities of CRISPR-based gene editing therapies. Can you summarize the main findings of your research and their implications for genetic therapies, especially for conditions like chronic granulomatosis?
Martin Jinek: Thank you for having me. Our study revealed both the promising and challenging aspects of CRISPR-based therapies. While we explored modified versions of CRISPR components and protective elements to improve safety, we found that none could entirely eliminate the risks of unwanted side effects. This highlights the need for continued research to develop safer and more effective gene editing approaches for hereditary diseases like chronic granulomatosis.
Editor: That’s insightful. What specific risks were identified during your research, and how do they affect the broader field of gene therapy?
Martin Jinek: The primary concern is the likelihood of the genetic scissors cutting chromosomes in multiple locations, which can lead to chromosomal rearrangements and other unintended genetic consequences. These risks pose significant challenges in the development of gene editing therapies. If we want to make CRISPR a reliable tool for treating genetic disorders, we must focus on minimizing these dangers while enhancing the precision of the technology.
Editor: With these challenges in mind, what advancements do you envision are necessary for CRISPR technology to become a safer option for patients?
Martin Jinek: We need further technological advancements that can enhance the precision of gene editing. This includes developing more advanced CRISPR components and methodologies that can target specific genomic loci with fewer off-target effects. Collaborative efforts between researchers in genetics, bioengineering, and clinical applications will be crucial. There’s also a need for rigorous testing protocols to ensure these new methods are both safe and effective.
Editor: What advice do you have for researchers or professionals in the field who are interested in pursuing CRISPR-based therapies?
Martin Jinek: My advice would be to remain patient and persistent. The promise of CRISPR technology is immense, but it requires a cautious approach. Engage with interdisciplinary teams to innovate and address safety and ethical challenges. Also, stay updated on the latest research findings, as this field is rapidly evolving. Collaborations and sharing insights will play a fundamental role in overcoming existing limitations.
Editor: As we look to the future, what do you believe will be the most significant milestones for CRISPR technologies in the next few years?
Martin Jinek: I believe we will see significant strides in precision editing, which will reduce off-target effects. Additionally, increased regulatory discussions surrounding the ethical implications of gene editing will pave the way for clearer guidelines in clinical applications. It’s also likely that we will better understand the long-term effects of CRISPR therapies through extensive clinical trials, which is essential for building public trust.
Editor: Thank you, Professor Jinek, for sharing your valuable insights. Your expertise is instrumental in navigating the future of genetic therapies and ensuring we harness the full potential of CRISPR technology while prioritizing safety.
Martin Jinek: Thank you for the opportunity to discuss this critical topic. The conversation on CRISPR and its implications for health is just beginning, and I look forward to seeing how it develops in the coming years.