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1980, ISBN 9788788043068, v.
Book
Human Mutation, ISSN 1059-7794, 01/1999, Volume 13, Issue 1, p. 83
  The infantile form of GSD II (an inherited deficiency of the lysosomal enzyme, acid ±-glucosidase, Pompe disease) is a severe and invariably fatal disease... 
Journal Article
Human Molecular Genetics, ISSN 0964-6906, 09/2001, Volume 10, Issue 19, p. 2039
  Both enzyme replacement and gene therapy of lysosomal storage disorders rely on the receptor-mediated uptake of lysosomal enzymes secreted by cells, and for... 
Journal Article
Journal Article
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2004, Volume 9, Issue S1, pp. S326 - S327
Gene therapies and recombinant enzyme replacement therapies (ERT) are currently under investigation as therapeutic options for several lysosomal storage... 
Journal Article
Surgical Oncology Clinics of North America, ISSN 1055-3207, 01/2008, Volume 17, Issue 1, pp. 219 - 232
Journal Article
Journal Article
Lancet, The, ISSN 0140-6736, 2008, Volume 371, Issue 9625, pp. 1695 - 1709
Journal Article
Lancet Oncology, The, ISSN 1470-2045, 2010, Volume 11, Issue 1, pp. 21 - 28
Journal Article