A breakthrough photothermal nanoblade has been successfully applied demonstrating mitochondria transfer into mammalian cells for mitochondrial disease therapeutics, regenerative medicine and stem cell therapy — that, according to NantWorks, LLC in collaboration with researchers at the UCLA Department of Pathology and Laboratory Medicine and Department of Mechanical and Aerospace Engineering.
Diseases associated with mitochondrial DNA mutations are prevalent, affecting on average one in 5,000 individuals. Tools for manipulating cells through mitochondrial transfer are limited and typically lack in efficiency.
The photothermal nanoblade utilizes a pulsed laser triggered at ultrafast speed enabling a cell membrane opening coupled with a pressure-driven cargo transport system to deliver extremely large cargo into cells. High transfer efficiency and high cell viability can be achieved for micron-sized cargo such as mitochondria. The commercial prototype, developed by NanoCAV, LLC, a NantWorks subsidiary, can perform high-throughput delivery into 100,000 cells in a few minutes.
The results of the technology were published in Cell Metabolism, which reported the successful transfer of wild-type mitochondria into cells deficient in normally functioning mitochondria, with restoration of mitochondrial function and metabolism profiles in the target cell after mitochondria transfer.
“The medical potential for this technology will help to enable new mitochondrial research and potential therapies for diseases,” said Patrick Soon-Shiong, MD, chairman and CEO of NantWorks. “Working with UCLA on this project has driven a prototype that is able to perform not only higher-throughput delivery but with an ease in usability for researchers.”
The technology was developed at UCLA in collaboration with NantWorks and led by Professors Michael Teitell and Eric Pei-Yu Chiou, Ting-Hsiang Wu, as well as Dr. Kayvan Niazi, scientist and chief technology officer (CTO) of NantBio, a wholly owned subsidiary of NantWorks.