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Type: Journal article
Title: Evaluation of fibroblast-mediated gene therapy in a feline model of mucopolysaccharidosis type VI
Author: Yogalingam, G.
Crawley, A.
Hopwood, J.
Anson, D.
Citation: BBA: General Subjects, 1999; 1453(2):284-296
Issue Date: 1999
ISSN: 0304-4165
Abstract: Fibroblast-mediated ex vivo gene therapy was evaluated in the N-acetylgalactosamine 4-sulfatase (4S) deficient mucopolysaccharidosis type VI (MPS VI) cat. Skin biopsies were obtained at birth from severely affected MPS VI kittens and used to initiate fibroblast outgrowths for retroviral transduction with the 4S cDNA. 4S gene expression in transduced cells was under the transcriptional control of the MoMLV long terminal repeat promoter or the cytomegalovirus (CMV) immediate-early promoter. Characterisation of gene-transduced fibroblasts demonstrated the cells to be over-expressing 4S activity. Twenty-four to forty million autologous, gene-corrected fibroblasts were implanted under the renal capsule of three MPS VI kittens at 8-16 weeks of age. Transient, low levels of 4S activity were detected in peripheral blood leukocytes shortly after implantation but were not detectable within 3-8 weeks' post-implantation. Long-term biochemical and clinical evaluation of these cats demonstrated identical disease progression to that previously described in untreated, clinically severe MPS VI cats.
Keywords: Kidney
Mucopolysaccharidosis VI
Disease Models, Animal
Skin Transplantation
Genetic Vectors
Genetic Therapy
DOI: 10.1016/S0925-4439(98)00112-4
Appears in Collections:Aurora harvest
Paediatrics publications

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