Novel therapeutics being developed for the treatment of childhood



spinal muscular atrophy (SMA) using Hamilton Microliter 700 syringes.

The laboratory of Professor Thomas Gillingwater at the University of Edinburgh is developing novel therapeutics for the treatment of the childhood neuromuscular disease, spinal muscular atrophy (SMA). As the genetic defects of SMA are well characterized, mouse models of SMA have been developed which are utilized to develop treatment strategies for this currently incurable condition.

In Professor Gillingwaters lab, Hamilton syringes and needles are used to accurately deliver small molecule compounds and gene therapy vectors via a variety of injection techniques (intravenous, intraperitoneal, intramuscular, subcutaneous, and intrauterine to the SMA mouse model and the potential therapeutic potential of a drug assessed by behavioral, histological and molecular analysis. This strategy has been employed to great success, with recent identification of novel pathways that can improve the SMA phenotype in animal models (Wishart et al., 2015, JCI; 124(4):1821-34; Powis et al., 2016 JCI Insight; 1(11):e87908).

Research projects in the Gillingwater lab, are mainly funded by a not-for-profit UK charity that supports research into this devastating condition: the SMA Trust (http://www.smatrust.org/).