Mentorship leads to new gene therapy discoveries

Pictured (L to R): Alex Davis; Lucie Guo, MD, PhD; Sui Wang, PhD; Stanley Qi, MA, PhD; and Jing Bian, PhD, are all collaborating on research focused on using CRISPR-based technology for ocular gene therapy.

Sui Wang, PhD, assistant professor of ophthalmology, attributes her career success to mentorship. While pursuing graduate school and her postdoctoral work, her mentors helped her lay a foundation for an independent research career in the future. During those years, Wang studied pancreatic development and diabetes, and retina cell specification, which looks at how different cell types are generated during development.

“I can testify to the major impact that mentorship played in my life professionally and personally and I wanted to pay it forward by mentoring the next generation at Stanford,” Wang said.

The Stanford Ophthalmology Advanced Research (SOAR) Residency Program provides department funding to residents to accelerate their academic research program by dedicating an extra year to full-time basic science or translational research. Lucie Guo, MD, PhD, is a SOAR resident co-mentored by Wang and Stanley Qi, MA, PhD, assistant professor of bioengineering and chemical and systems biology. Guo’s research focuses on developing innovative Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-based technology for ocular gene therapy.

I can testify to the major impact that mentorship played in my life professionally and personally and I wanted to pay it forward by mentoring the next generation at Stanford.

“CRISPR technology can alter DNA, which could possibly block or delay the disease initiation at a very early stage, helping patients’ quality of life and possibly treat disease,” Wang said.

Through protein engineering in test tubes, they developed an optimized CRISPR enzyme called vgCas12a which has powerful capabilities of gene editing, activation and repression. Then, they delivered this new CRISPR enzyme by subretinal injection in mice and saw that it drove therapeutic gene regulation, with wide implications for regenerative medicine in the retina. They received a $100,000 grant through the 2021 Stanford-Coulter Translational Research Grant to further advance this technology.

“I’m thankful for Dr. Wang’s mentorship as a world expert in retina biology, and Dr. Qi’s mentorship as a CRISPR pioneer, as well as our teammates and collaborators,” Guo said. “The SOAR program was the perfect incubator for this interdisciplinary project that bridges ophthalmology and the larger scientific community at Stanford.”



By KATHRYN SILL

Kathryn Sill is a web and communications specialist for the Byers Eye Institute in the Department of Ophthalmology, at Stanford University School of Medicine. Email her at ksill@stanford.edu.