Gretchen Ertl/Whitehead Institute
Aditya Raguram
In our lab, we’re tackling the “protein delivery problem:” how to get large molecules like proteins and RNAs into living cells. These molecules include genome-editing tools that can target and correct mutations in DNA to address the root cause of genetic diseases. But cells are like fortresses, making it very hard for these large molecules to get inside.
To solve this problem, we’re especially excited about using biological nanoparticles, tiny particles that many cells naturally release. If we can understand how cells make these particles and load them with the protein or RNA “cargo” we want to deliver, we can harness them to safely and efficiently deliver genome-editing tools.
Instead of testing each particle individually, we’ve developed methods to study thousands, or even millions, of particle variants at once. By examining how particle design, cargo type, and the producing cell influence particle production and delivery, we’re learning what makes a particle most effective.
By combining this information across particle types and cargos, we aim to create a complete roadmap for engineering these nanoparticles. The ultimate goal is to reliably deliver genome-editing tools and other large therapies into human cells, opening the door to new treatments for genetic disorders and potentially other diseases.
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