Welcome to the Kay Lab
Pinoeering Gene Therapy and Non-Coding RNA Discoveries at Stanford
Our laboratory has a long-standing interest in the development of gene transfer vectors for gene therapy and the manipulation of non-coding RNAs for therapeutic purposes. A major focus involves unraveling the mechanisms of viral transduction in vivo, particularly with mini-circles and recombinant AAVs (rAAV). This foundational work aims to uncover key mechanistic insights, ultimately advancing the potential of gene transfer vectors to treat human disease.
...advancing the potential of gene transfer vectors to treat human disease.
Our Research Focus
Here is a glimpse into our primary research endeavors:
Gene Transfer & AAV Biology
Our work specifically focuses on the development of gene transfer vectors for gene therapy, with a major interest in unraveling the mechanism of viral vector transduction in vivo. We particularly leverage two key vector systems: mini-circles and recombinant AAVs (rAAV). Understanding how these AAV capsids function is crucial for evaluating their clinical efficacy and resolving current mysteries in gene therapy trials.
Non-Coding RNA Therapeutics
We are deeply invested in manipulating non-coding RNAs for therapeutic purposes. Our lab has uncovered key mechanistic insights into how non-coding miRNAs are loaded into active RISC complexes in mammals. We also discovered new classes of small RNAs derived from tRNA and non-coding RNAs that may be generated by RNA-directed RNA transcription in vertebrates.
Visit Stanfords Pediatric Division of Human Gene Therapy
The Kay Lab is part of the Division of Human Gene Therapy at Stanford's School of Medicine, which leads interdiciplinary efforts in developing and translating genetic therapies for human disease. Learn more about the division's clinical programs, broader research initiatives, and collaborative work.
