000 02872nam a22005055i 4500
001 978-1-4614-8357-1
003 DE-He213
005 20140220082831.0
007 cr nn 008mamaa
008 131010s2013 xxu| s |||| 0|eng d
020 _a9781461483571
_9978-1-4614-8357-1
024 7 _a10.1007/978-1-4614-8357-1
_2doi
050 4 _aQH588.S83
072 7 _aPSAK
_2bicssc
072 7 _aSCI017000
_2bisacsh
072 7 _aSCI049000
_2bisacsh
082 0 4 _a571.6
_223
100 1 _aBoelens, Jaap Jan.
_eeditor.
245 1 0 _aStem Cell Therapy in Lysosomal Storage Diseases
_h[electronic resource] /
_cedited by Jaap Jan Boelens, Robert Wynn.
264 1 _aNew York, NY :
_bSpringer New York :
_bImprint: Humana Press,
_c2013.
300 _aIX, 171 p. 6 illus., 3 illus. in color.
_bonline resource.
336 _atext
_btxt
_2rdacontent
337 _acomputer
_bc
_2rdamedia
338 _aonline resource
_bcr
_2rdacarrier
347 _atext file
_bPDF
_2rda
490 1 _aStem Cell Biology and Regenerative Medicine,
_x2196-8985
505 0 _a1. Lysosomal Diseases and Therapeutic Options: An Overview -- 2. Alternative Treatment Options: Enzyme Replacement and Small Molecule Therapies -- 3. Hematopoietic Cell Transplantation in Inborn Errors of Metabolism -- 4. Allogeneic Hematopoietic Stem Cell Transplantation in Lysosomal Storage Disorders: Long Term Neurodevelopmental and Somatic Outcome -- 5. Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders: Expected Benefits and Limitations -- 6. Alternative Future Therapies for Lysosomal Storage Diseases: Embryonic Stem Cell- and Induced Pluripotent Stem Cell Therapy -- 7. New Directions and Conclusions.
520 _aStem Cell therapy for lysosomal diseases (LSDs) is developing rapidly. This volume discusses the history, current practice and future perspectives of stem cells in inborn errors of metabolism (IEM) and provides an international perspective on progress, limitations, and future directions (e.g. gene therapy, iPS, ES) in the field. Beginning with an overview of these diseases, the book covers the breadth of this topic from treatment options, bone marrow transplantation, and alternative treatment options, through long-term outcomes and future perspectives.
650 0 _aLife sciences.
650 0 _aHuman genetics.
650 0 _aCytology.
650 0 _aStem cells.
650 1 4 _aLife Sciences.
650 2 4 _aStem Cells.
650 2 4 _aCell Biology.
650 2 4 _aHuman Genetics.
700 1 _aWynn, Robert.
_eeditor.
710 2 _aSpringerLink (Online service)
773 0 _tSpringer eBooks
776 0 8 _iPrinted edition:
_z9781461483564
830 0 _aStem Cell Biology and Regenerative Medicine,
_x2196-8985
856 4 0 _uhttp://dx.doi.org/10.1007/978-1-4614-8357-1
912 _aZDB-2-SBL
999 _c96048
_d96048