The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update
Author:
Publisher
Springer Science and Business Media LLC
Subject
Transplantation,Hematology
Link
http://www.nature.com/articles/bmt200862.pdf
Reference19 articles.
1. Vellodi A . Lysosomal storage disorders. Br J Haematol 2004; 128: 413–431.
2. Beck M . New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet 2007; 121: 1–22.
3. Proia RL, Wu YP . Blood to brain to the rescue. J Clin Invest 2004; 113: 1108–1110.
4. Peters C, Steward CG . Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 2003; 31: 229–239.
5. Rovelli AM, Steward CG . Hematopoietic stem cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions. Bone Marrow Transplant 2005; 35: S23–S26.
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