Stanford Scientists Accelerate CRISPR Innovations to Develop Life-Saving Treatments Faster
For many people, waiting is simply not an option, especially when it comes to treating life-threatening diseases. In the "Research Matters" series, we visit labs across Stanford’s campus to hear from scientists about their ongoing work, its potential impact on human health and well-being, and the vital role universities play in driving national innovation. This summary captures the essence of what these researchers are sharing. Our goal is straightforward: transform groundbreaking science into practical solutions that save lives. In my lab, we have been engineering human immune cells to enhance their ability to combat tumors. We are also exploring ways to delay the onset of neurological aging, a process that leads to symptoms such as memory loss, cognitive decline, and impaired motor function. These conditions often appear later in life but often too soon. Imagine if we could push them back by another 50 years. My ultimate vision is to develop a genetic medicine that can be safely introduced into the brain to halt Alzheimer’s disease. Over the past five years, my team has been pioneering methods to improve the safety and efficiency of CRISPR, a gene-editing tool derived from bacterial immune systems. We have engineered CRISPR to be smaller, more precise, and safer for delivery into human cells and tissues. To foster broader advancements, we have made our CRISPR tools widely available to other researchers, who are using them to address Parkinson’s, liver disease, lung disease, and heart disease. Our tools have been shared over 10,000 times, demonstrating the collaborative nature of scientific progress. When I entered the field of CRISPR in 2013, the idea of using it in human cells was speculative. It was surprising and encouraging to see that within just 10 years, Casgevy, a CRISPR-based treatment for sickle cell anemia, received FDA approval. However, as I reflect on this achievement, I realize that 10 years is far too long. With the technological advancements of 2025—such as automation and AI—I believe we can accelerate a decade’s worth of work in half the time. The urgency of our mission became particularly clear after we published our initial findings. We received numerous messages from patients and their families eager to access our treatments. At that time, we had to politely inform them that we were still in the early stages of development. For many of these individuals, the wait was intolerable. This realization has spurred us to shift our focus from merely creating tools to actively translating these innovations into viable therapies. This transition demands extensive collaboration with experts from various fields and the biotech industry. One of our compact CRISPR tools has already progressed to clinical trials for treating a form of muscular dystrophy, a journey that took only three years—a fraction of the usual timeframe, which typically exceeds six years. Despite these significant strides, I often feel we are not doing enough. Each day, the need for rapid progress intensifies, and we must redouble our efforts. Science, much like a chemical reaction, requires time to mature. Sustained commitment is crucial, especially in complex areas like biomedical and disease research, where success often depends on cumulative knowledge and expertise. If we falter in training the next generation of scientists, we risk losing our edge in a highly competitive global landscape, even if we have the most promising solutions. Fortunately, scientists are adept at overcoming challenges. This resilience is a hallmark of our training and reflects the innovative spirit at Stanford. We are committed to tackling the most daunting scientific problems and will not be deterred by setbacks. Whenever I face moments of doubt, I remind myself of our mission: to develop life-saving treatments for those battling life-threatening diseases. This mission fuels our drive to push the boundaries of what is possible and bring hope to those who need it the most.
