Developing Novel Therapeutics for Childhood Spinal Muscular Atrophy

Date: October 2016
University of Edinburgh | Neuroscience
Thomas Gallingwater

Novel therapeutics are 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 Gillingwater's 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/).

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