Medical Applications of Synthetic Biology

Genetically engineered mammalian cells represent the next frontier in medicine.  Unique to cell-based platforms is their ability to perform highly robust logical operations by sensing and responding to multiple stimuli in vivo, which can revolutionize medicine by creating advanced cell and gene therapies, tissues/organs, vaccines, and diagnostics.  However, despite recent advances in genetic engineering and genome editing, the technological barrier to creating user-defined mammalian cell functions remains immense.  To obtain the necessary precision required for complex spatiotemporal control of cell function, we are embarking on an ambitious, design-driven research program to develop innovative technologies for next-generation therapeutics and diagnostics, with applications in cancer, autoimmunity, vaccines, and diabetes.

New Nature Biotechnology Paper: Substitution of modified nucleotides in self-amplifying RNA (saRNA) suppresses interferon response while maintaining translation efficacy

By Arun NambiarJuly 8th, 2024

A huge congratulations to Josh on the publication of his work on saRNA in Nature Biotech this morning!

saRNA is an exciting platform for delivering genetically-encoded therapeutics, and the discovery of replicon-compatible modified nucleotides for enhanced translation efficiency and minimal immunogenicity opens the door for it's use across a broad stretch of applications: from vaccines as demonstrated in this publication, to the immunotherapy, protein-replacement therapy, and cell therapy spaces as well.

This work has opened up an exciting new avenue for our work in the Wong Lab. Congrats again to Josh, and all the other team members from the Wong, Grinstaff and Douam labs!

https://www.nature.com/articles/s41587-024-02306-z