Purpose of the STSM: Spinal cord injury (SCI) is a devastating condition that affects peripheral and central nervous system (PNS and CNS, respectively) and often leads to permanent functional and neurological deficits. The team from the initial laboratory developed a new family of fully biodegradable dendrimers to act as nucleic acid carriers to promote the downregulation of PTEN expression, which showed to play a role in cell death surrounding injury. Dendrimers showed good ability to complex siRNA (≥75%) and the resulting dendriplexes (dendrimer-siRNA complexes) showed low nanosizes (≤60 nm), spherical morphologies and positive surface charge. Furthermore, dendriplexes showed no toxic effects in the neuronal cells (cell lines, primary cultures and ex vivo model). Neurotargeting ability of our dendriplexes was already confirmed through flow cytometry and atomic force microscopy.
Additionally, they developed a new microfluidic model mimicking the interface between neuromuscular junction (NMJ), PNS and CNS neural cells. This alternative model was crucial to evaluate dendriplexes trafficking pathway and its capacity to reach CNS.
With this STSM, they wanted to pursue exploring that delivery vector and enhancing their microfluidic model. Therefore, the goal for this STSM was to evaluate dendriplexes’ electrophysiological effects in neurons through the integration of microelectrode arrays (MEAs) in our microfluidics.

Grantee name: Ana Spencer

STSM start and end date: 2023-04-13 to 2023-04-22

Title: Targeted neuronal nucleic acid vectors based on fully biodegradable dendrimers as an
innovative treatment for spinal cord injury
Host lab: Dr. Ben Maoz, Dep. of Bio-Medical Engineering, Tel Aviv University (Tel Aviv, Israel)