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  • MG-110 Intravenous neonatal gene therapy corrects GM2 gangliosidoses in sandhoff mice for ‘long-term’, by using an aav expressing a new hexosaminidase variant

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Clinical Genetics
Posters MG-100 to MG-119
MG-110 Intravenous neonatal gene therapy corrects GM2 gangliosidoses in sandhoff mice for ‘long-term’, by using an aav expressing a new hexosaminidase variant

Authors

  • Karlaina JL Osmon 1Centre for Neuroscience Research, Queen’s University, Kingston, ON, Canada, K7L 3N6 PubMed articlesGoogle scholar articles
  • Evan Woodley 2Department of Biomedical and Molecular Sciences, Queen’s University, Kingston, ON, Canada, K7L 3N6 PubMed articlesGoogle scholar articles
  • Patrick Thompson 3Medical Genetics/Departments of Pediatrics and Pathology and Molecular Medicine, Queen’s University, Kingston, ON, Canada, K7L 2V7 PubMed articlesGoogle scholar articles
  • Katalina Ong 3Medical Genetics/Departments of Pediatrics and Pathology and Molecular Medicine, Queen’s University, Kingston, ON, Canada, K7L 2V7 PubMed articlesGoogle scholar articles
  • Subha Karumuthil-Melethil 4Gene Therapy Centre, University of North Carolina, Chapel Hill, NC, USA PubMed articlesGoogle scholar articles
  • Brian Mark 5Department of Microbiology, University of Manitoba, Winnepeg, Manitoba, Canada, R3T 2N2 PubMed articlesGoogle scholar articles
  • Don Mahuran 6Genetics and Genome Biology, Sick Kids, Toronto, ON, Canada, M5G 0A4 7Department of Laboratory Medicine and Pathology, University of Toronto, Toronto, ON, Canada, M5S 1A8 PubMed articlesGoogle scholar articles
  • Steven J Gray 4Gene Therapy Centre, University of North Carolina, Chapel Hill, NC, USA 8Department of Ophthalmology, University of North Carolina, Chapel Hill, NC, USA PubMed articlesGoogle scholar articles
  • Jagdeep S Walia 1Centre for Neuroscience Research, Queen’s University, Kingston, ON, Canada, K7L 3N6 2Department of Biomedical and Molecular Sciences, Queen’s University, Kingston, ON, Canada, K7L 3N6 3Medical Genetics/Departments of Pediatrics and Pathology and Molecular Medicine, Queen’s University, Kingston, ON, Canada, K7L 2V7 PubMed articlesGoogle scholar articles

Citation

Osmon KJ, Woodley E, Thompson P, et al
MG-110 Intravenous neonatal gene therapy corrects GM2 gangliosidoses in sandhoff mice for ‘long-term’, by using an aav expressing a new hexosaminidase variant
Journal of Medical Genetics 2015;52:A4.

Publication history

  • First published December 4, 2015.
Online issue publication 
April 27, 2016

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© 2015, Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions