Research Topics
Species | S W J McPheeSummaryAffiliation: New Jersey Country: USA Publications
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Detail Information
Publications
Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan diseaseS W J McPhee
Cell and Gene Therapy Center, UMDNJ Robert Wood Johnson Medical School, 401 Haddon Avenue, Suite 395, Camden, NJ 08103, USA
Brain Res Mol Brain Res 135:112-21. 2005..This study provides evidence that AAV-2-mediated aspartoacylase gene transfer to the brain improves biochemical and behavioral deficits in tremor rat mutants (tm/tm) and supports the rationale of human gene transfer for Canavan disease...
Immune responses to AAV in a phase I study for Canavan diseaseS W J McPhee
Department of Surgery, Division of Neurosurgery, Robert Wood Johnson Medical School, University of Medicine and Dentistry of New Jersey, 401 Haddon Avenue, Camden, NJ 08103, USA
J Gene Med 8:577-88. 2006..Long-term monitoring of subjects and expansion to phase II/III will be necessary in order to make definitive statements on safety and efficacy...
Natural history of Canavan disease revealed by proton magnetic resonance spectroscopy (1H-MRS) and diffusion-weighted MRIC G Janson
UMDNJ Robert Wood Johnson Medical School, Dept of Neurosurgery, Camden, NJ 08103, USA
Neuropediatrics 37:209-21. 2006..The resultant model can be used to non-invasively monitor the natural history of Canavan disease or related leukodystrophies in future studies involving drug, gene therapy, or stem cell treatments...
