‘For many people, waiting is not an option’

In the “Research Matters” series, we visit labs across campus to hear directly from Stanford scientists about what they’re working on, how it could advance human health and well-being, and why universities are critical players in the nation’s innovation ecosystem. The following are the researchers’ own words, edited and condensed for clarity.

Our vision is simple: Turn groundbreaking science into real solutions that save lives. We’ve worked on engineering human immune cells to make them better defenders against tumors. We’re also interested in engineering genetics to delay the onset of neurological aging. Symptoms like memory loss, decline in cognitive skills, or impaired motor function typically occur in a later stage of life – but not late enough. What if we could delay them by another 50 years?

My dream is to make a genetic medicine that we can safely introduce to the brain to completely stop Alzheimer’s.

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Researchers develop a hypercompact CRISPR

Bioengineers have repurposed a “non-working” CRISPR system to make a smaller version of the genome engineering tool. Its diminutive size should make it easier to deliver into human cells, tissues and the body for gene therapy.

My lab has spent the last five years innovating how CRISPR, a gene-editing tool, can be used safely in the human body. Because the technology leverages an immune system from bacteria, we’ve had to engineer it to make it smaller, more efficient, and safely deliverable into human cells and tissues.

We also make sure our CRISPR tools are broadly available. Other researchers are using them to address Parkinson’s, liver disease, lung disease, and heart disease. We’ve shared our tools with other researchers more than 10,000 times.

When I started in the field of CRISPR in 2013, we didn’t even know it could work in human cells. No one imagined it would lead to an approved drug – Casgevy, a treatment for sickle cell anemia – only 10 years later. That’s pretty impressive.

But now I look back and I think, 10 years is not good enough. Given what we have in 2025 – automation, AI, and many new tools – I feel it’s possible to do another decade’s worth of work in half that time.

When we published our initial studies, we got many emails from patients and their families asking when they could access our treatments. At the time, I had to tell them, “Sorry, but we are still in an early stage.”

But for many of these people, waiting is not an option. Realizing this urgency, we’ve gradually switched our focus from purely developing tools to actively translating innovations into real therapies, which requires a lot of collaboration with experts from other fields and the biotech sector.

In fact, one of our compact CRISPR tools has already entered clinical trials for treating a type of muscular dystrophy, moving from lab discovery to patient treatment in just three years – a process that typically takes more than six.

Despite our progress, I constantly feel we haven’t done enough. We need to do more. And for that, we need continuous support.

Science requires sustained commitment because projects, particularly in biomedical and disease research, require an accumulation of knowledge and training. Research is like a chemical reaction – it takes time to reach completion. If we ever slow down in training the next generation of scientists, we risk falling behind other competitors in the world – even if we have the better solution.

Fortunately, scientists are good at addressing challenges. That’s what we trained for, and it’s part of the spirit of Stanford: We tackle the most challenging scientific problems, and we will not be discouraged by failures or adversities. When I have doubts, I think about my mission: developing life-saving treatments for those facing life-threatening diseases.