1. 014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update. (29th July 2019) Authors: Farrar, Michelle; Swoboda, Kathryn J; Schultz, Meredith; McMillan, Hugh; Parsons, Julie; Alexander, Ian E; Kernbauer, Elaine; Farrow, Marcia; Ogrinc, Francis G; Feltner, Douglas E; McGill, Bryan E; Spector, Sidney A; L'Italien, James; Sproule, Douglas M; Strauss, Kevin A Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 90(2019)e7 Page Start: A5 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Great expectations: virus-mediated gene therapy in neurological disorders. Issue 8 (5th June 2020) Authors: Kariyawasam, Didu; Alexander, Ian E; Kurian, Manju; Farrar, Michelle Anne Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 91:Issue 8(2020) Page Start: 849 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease. (12th September 2019) Authors: McNamara, Elyshia L; Taylor, Rhonda L; Clayton, Joshua S; Goullee, Hayley; Dilworth, Kimberley L; Pinós, Tomàs; Brull, Astrid; Alexander, Ian E; Lisowski, Leszek; Ravenscroft, Gianina; Laing, Nigel G; Nowak, Kristen J Journal: Human molecular genetics Issue: Volume 29:Number 1(2020) Page Start: 20 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗