alexa Report: Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies
ISSN: 2157-7412

Journal of Genetic Syndromes & Gene Therapy
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Conference Proceeding

Report: Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies

Anthony Blaeser1*, Amy Harper1, Kevin Campbell2 and Qi Long Lu1

1McColl-Lockwood Laboratory for Muscular Dystrophy Research, Neuromuscular/ALS Center and Clinical Genetics/Department of Pediatrics, Levine Children’s Hospital,Carolinas Medical Center, Carolinas HealthCare System, Charlotte, NC, USA

2Howard Hughes Medical Center, Roy J and Lucille A Carver College of Medicine, The University of Iowa, Iowa City, IA, USA

*Corresponding Author:
Anthony Blaeser
PhD, Carolinas HealthCare System, 1000 Blythe Blvd, Charlotte
NC 28203, United States
Tel: 7043554652
E-mail: [email protected]

Received date: January 11, 2016; Accepted date: February 05, 2016; Published date: February 12, 2016

Citation: Blaeser A, Harper A, Campbell K, Lu QL (2016) Report: Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies. J Genet Syndr Gene Ther 7:286. doi:10.4172/2157-7412.1000286

Copyright: © 2016 Blaeser A, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and

 

Abstract

The Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies took place on April 16- 17, 2015 at the Fairfield Inn and Suites Charlotte Uptown, Charlotte, North Carolina. The workshop was hosted by the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and sponsored by the Carolinas HealthCare Foundation, the Muscular Dystrophy Association (MDA), funds raised by “Riding 4 Research” and generous support from the McColl and Lockwood families. Clinicians and scientists from the US, UK, Germany and Japan presented a total of 21 talks spread out over 2 days. The workshop was divided into three sessions: Session A focused on the current status of the development of animal models for diseases caused by defects in muscle-protein glycosylation, on our current understanding of such glycosylation and on the mechanisms that lead to disease. Session B focused on preclinical therapeutics, in particular on AAV- and drug-based therapies. Session C covered the clinical management of muscular dystrophies and endpoint evaluation.

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