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Type: Thesis
Title: Enzyme-replacement therapy in lysosomal storage diseases / Julie Bielicki.
Author: Bielicki, Julie
Issue Date: 2003
School/Discipline: Dept. of Paediatrics
Abstract: Describes experiments involving two animal models; the MPS VI cat and the fucosidosis dog to demonstrate the effectiveness of enzyme-replacement therapy (ERT) in lysosomal storage diseases (LSD). Pathology in these animal models closely parallels that observed in the human condition. In terms of therapeutic strategies, the results described in this thesis indicate that the use of same-species enzyme is superior to non-same-species enzyme. ERT used on the fucosidosis dog appeared to delay the onset of neurological symptoms. Although technically difficult to produce, chimeric proteins may provide a means of treatment for neurological symptoms in LSD.
Dissertation Note: Thesis (Ph.D.)--University of Adelaide, Dept. of Paediatrics, 2003
Description: Bibliography: leaves 266-289.
xix, 289 leaves : ill. (some col.), plates (some col.) ; 30 cm.
Provenance: This electronic version is made publicly available by the University of Adelaide in accordance with its open access policy for student theses. Copyright in this thesis remains with the author. This thesis may incorporate third party material which has been used by the author pursuant to Fair Dealing exception. If you are the author of this thesis and do not wish it to be made publicly available or If you are the owner of any included third party copyright material you wish to be removed from this electronic version, please complete the take down form located at:
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