UCLA Researchers in the Departments of Chemistry and Materials Science & Engineering have developed a novel means of delivering intracellular cargo.
Gene editing of various cell types remains the focus of many therapeutic approaches across a wide spectrum of disease states. However, intracellular delivery of gene editing material, or other cargo, to the most exciting target cell types (e.g. stem and immune cells) remains difficult. Various approaches for intracellular delivery have been developed based broadly on one of two approaches: membrane-disruption-mediated or carrier-mediated delivery. Unfortunately, current techniques such as sonoporation suffer from low efficiency of gene transfer and high cell death due to irreversible damage to the cell membrane, all while requiring cells be fixed in one place during cargo delivery. An ideal technology for intracellular cargo delivery would cause minimal cell perturbation, be scalable, be useful across all cell types and be cost effective.
Dr. Weiss and collaborators are developing a novel microfluidics device which utilizes vibrational cell deformation to deliver intracellular cargo. The invention both aligns cells for single cell flow-through and delivers cargo intracellularly by deforming the target cell membrane. Use of vibrational cell deformation within a microfluidic device removes the fouling associated with other devices where compressive force is applied via physical contact with microchannel walls. This invention is designed to be scalable via use in a highly parallel setting and is compatible with GMP processes. As proposed this novel device could allow for genetic manipulation of cells necessary for hematopoietic stem cell transplant for an infant in 1 hour.
The device concept has been proposed, with a prototype in current development.
|European Patent Office||Published Application||3580556||12/18/2019||2017-454|
Additional Patent Pending
acoustic waves, intracellular cargo, cargo delivery, transduction, expression, microfluidic, transfection