Long-term correction of rat model of Parkinson's disease by gene therapy
Article Abstract:
Rat muscle cells were lipofected by a tyrosine hydroxylase expression plasmid and used for intracerebrla transplantation into rat models of Parkinson's disease. The transplanted cells continued to express tyrosine hydroxylase during the six-month duration of the experiment. In addition, the mean contralateral rotations induced by apomorphine decreased significantly by 75% for at least six months after transplantation. These results indicate that transplantation of muscle cells expressing tyrosine hydroxylase can promote a long-term correction of Parkinson's disease in a rat model system.
Publication Name: Nature
Subject: Zoology and wildlife conservation
ISSN: 0028-0836
Year: 1993
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Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer
Article Abstract:
Lysosomal storage in the spleen and liver, associated with mucopolysaccharidosis (Sly syndrome), can be mitigated by gene therapy in mice. A beta-glucuronidase gene is transferred into mutant hematopoietic stem cells through the mediation of a retroviral vector. This results in the low-level, long-term expression of beta-glucuronidase, reducing lysosomal accumulation in the spleen and liver and increasing the life span of the treated mice.
Publication Name: Nature
Subject: Zoology and wildlife conservation
ISSN: 0028-0836
Year: 1992
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