Developing new therapies for motor neurone disease (SMA and ALS)

About the project

Work in our laboratory is trying to identify new and effective ways to protect motor neurons (and other affected cells and tissues of the body) against disease in both childhood and adult forms of MND. A lot of our focus is currently on spinal muscular atrophy (SMA), where we are using both drug-based and gene therapy-based approaches to target key disease pathways. In particular, we are focussing on so-called ‘combinatorial therapies’ where you bring together multiple treatments in order to get maximum clinical benefit. We are also exploring how therapies developed for SMA may be applicable to help patients with ALS.


UK SMA Research Consortium (SMA Trust); SMA Europe; MND Scotland; Wellcome Trust; Medical Research Council; Anatomical Society


Groen EJN, Talbot K, Gillingwater TH
Advances in therapy for spinal muscular atrophy: promises and challenges
Nat Rev Neurol. 2018 Apr;14(4):214-224
Bernabò P, Tebaldi T, Groen EJN, Lane FM, Perenthaler E, Mattedi F, Newbery HJ, Zhou H, Zuccotti P, Potrich V, Shorrock HK, Muntoni F, Quattrone A*, Gillingwater TH*, Viero G* (*joint senior/corresponding authors)
In Vivo Translatome Profiling in Spinal Muscular Atrophy Reveals a Role for SMN Protein in Ribosome Biology
Cell Rep. 2017 Oct 24;21(4):953-965
Wishart TM, Mutsaers CA, Riessland M, Reimer MM, Hunter G, Hannam ML, Eaton SL, Fuller HR, Roche SL, Somers E, Morse R, Young PJ, Lamont DJ, Hammerschmidt M, Joshi A, Hohenstein P, Morris GE, Parson SH
Skehel PA, Becker T, Robinson IM, Becker CG, Wirth B, Gillingwater TH
Dysregulation of ubiquitin homeostasis and β-catenin signaling promote spinal muscular atrophy
J Clin Invest. 2014 Apr;124(4):1821-34

Primary location


Principal Investigator

Other people involved

Dr Ewout Groen

Dr Helena Chaytow

Dr Nikky Huang

Dr Kiterie Faller

Ines Boehm

Leire Ledahawsky

Dinja van der Hoorn