The ENDURE-LA: AAV Neuroscience Training Program is funded by a $1.67M grant and led by Professor Andrew Steele. It's part of NIH’s Brain Endure initiative to increase opportunities for students to pursue Ph.D.s in neuroscience. The need is clear, with demand for neuroscience researchers growing at a rate of around 12 % over the next decade.
This two-year program will provide CPP students with a year of neuroscience research training in Steele’s lab with a focus on adeno-associated virus (AAV) production and characterization. The lab, which includes a vector core and produces vectors for other research institutions, gained expertise in AAV production through a previous partnership with Caltech. AAV are used because they’re non-pathogenic and can cross the blood/brain barrier. The lab is engineering strains to target specific neurons.
“We fully control the genome and have a viral core and the infrastructure needed to produce at a large scale,” Steele said.
Senior Renqi Wu, who has been in Steele’s lab for about a year, shared, “I have gained profound understanding and experience utilizing a viral approach to target specific subpopulations of neurons to either express a fluorescent reporter, both knockout and rescue a gene (tyrosine hydroxylase), or to induce an ablation.”
A new course was developed that will be included in the year of training at CPP. The new course, Applications of Gene Therapy to Study the Brain and Treat Brain Disorders, will provide students with a foundation in gene therapy techniques and their use in neuroscience research with an AAV focus.
After their year of training, students will have a full-time summer research experience at UCLA in an NIH-supported lab where they’ll contribute to ongoing neuroscience research. They will learn advanced neuroscience techniques and approaches, such as in vivo imaging, electrophysiology, and optogenetics. During the following academic year, students will continue to support UCLA projects with viral production, optimization, or tissue processing.
Senior Sawyer Hardy plans to pursue a Ph.D. in molecular biology. “I’m interested in this research because I want to learn more about the current technologies being developed for genetic access and how they can be used to open an entirely new realm of scientific questions,” they said.
“I have gotten to learn and practice more in vitro work (using cell culture). I’ve learned more about how recombinases and genetic access tools work and how to develop them to be useful technologies in research. I also learned how these recombinase tools and other techniques are used to map neural circuits, and many of the things that researchers are learning using these discoveries.” Hardy’s ultimate goal is to perform clinical research focusing on gene therapy and genetic access technologies.
Professor Steele recently received a supplemental NIH grant for a widefield fluorescence microscopy system. “It’s a first step toward whole brain imaging,” he said.
“Students will gain technical expertise, and build professional networks, empowering them to pursue advanced degrees. This immersive experience will integrate students into the broader neuroscience research community, inspire long-term engagement, and cultivate meaningful mentorship relationships. By fostering a well-trained cohort of neuroscience researchers, this partnership with UCLA creates a sustainable model for promoting excellence in STEM education and research.”