Lentiviral Stem Cell Gene Therapy for Pompe Disease

Issue title: Abstracts from the 7th European Symposium on Steps Forward in Pompe Disease: Turin, Italy, November 21–22, 2014

Article type: Research Article

Authors: Liang, Qiushi | Stok, Merel | van Helsdingen, Yvette | van der Velden, Guus | Jacobs, Ed | Duncker, Dirk | Reuser, Arnold | van der Ploeg, Ans | Vulto, Arnold | van Til, Niek P. | Wagemaker, Gerard

Affiliations: Department of Hematology, Erasmus MC University Medical Center, Rotterdam, Netherlands | Center for Lysosomal and Metabolic Diseases & Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, Netherlands | Department of Genetics, Erasmus MC University Medical Center, Rotterdam, Netherlands | Department of Experimental Cardiology, Erasmus MC University Medical Center, Rotterdam, Netherlands | Hospital Pharmacy, Erasmus MC University Medical Center, Rotterdam, Netherlands | Hacettepe University, Stem Cell Research and Development Center, Ankara, Turkey

Note: [] Correspondence to: Gerard Wagemaker, Hacettepe University, Stem Cell Research and Development Center, 06100 Sihhiye, Ankara, Turkey. E-mail: [email protected]

DOI: 10.3233/JND-159055

Journal: Journal of Neuromuscular Diseases, vol. 2, no. s1, pp. S64-S64, 2015