Affiliation: New Jersey
- Long-term follow-up after gene therapy for canavan diseasePaola Leone
Department of Cell Biology, Cell and Gene Therapy Center, University of Medicine and Dentistry of New Jersey, Stratford, NJ 08034, USA
Sci Transl Med 4:165ra163. 2012..AAV2-ASPA gene therapy resulted in a decrease in elevated NAA in the brain and slowed progression of brain atrophy, with some improvement in seizure frequency and with stabilization of overall clinical status...
- Aspartoacylase supports oxidative energy metabolism during myelinationJeremy S Francis
Cell and Gene Therapy Center, Department of Cell Biology, University of Medicine and Dentistry of NJ, Stratford, New Jersey 08084, USA
J Cereb Blood Flow Metab 32:1725-36. 2012..N-acetylaspartic acid is proposed here to support lipid synthesis and energy metabolism via the provision of substrate for both cellular processes during early postnatal development...
- Transcriptional regulation of N-acetylaspartate metabolism in the 5xFAD model of Alzheimer's disease: Evidence for neuron-glia communication during energetic crisisSamantha Zaroff
Cell and Gene Therapy Centre, Department of Cell Biology, Rowan University School of Osteopathic Medicine, Stratford, NJ, USA Graduate School of Biomedical Sciences, Rowan University School of Osteopathic Medicine, Stratford, NJ, USA
Mol Cell Neurosci 65:143-52. 2015..This mechanism is proposed to be a fundamental means by which the brain conserves available substrate during energy crises. ..
- Dietary triheptanoin rescues oligodendrocyte loss, dysmyelination and motor function in the nur7 mouse model of Canavan diseaseJeremy S Francis
Cell and Gene Therapy Center, Department of Cell Biology, Rowan University School of Osteopathic Medicine, 40 East Laurel Rd, Stratford, NJ, USA
J Inherit Metab Dis 37:369-81. 2014..These results support the targeting of oxidative integrity at early stages of Canavan disease, and provide a foundation for the clinical development of a non-invasive dietary triheptanoin treatment regimen. ..
- Serial 1H-MRS in GM2 gangliosidosesMitra Assadi
Division of Neurology, University of Medicine and Dentistry of New Jersey, 3 Cooper Plaza, Suite 320, Camden, NJ 08103, USA
Eur J Pediatr 167:347-52. 2008..1H-MRS may be a valuable tool in monitoring the disease progress and response to therapy in GM2 gangliosidoses. Elevation in the mI may prove to be more sensitive than the other metabolite alterations...
- Endogenous aspartoacylase expression is responsive to glutamatergic activity in vitro and in vivoJeremy S Francis
Cell and Gene Therapy Center, UMDNJ School of Osteopathic Medicine, Stratford, New Jersey, USA
Glia 59:1435-46. 2011....
- Aspartoacylase gene transfer in brain/Canavan DiseasePaola Leone; Fiscal Year: 2004..abstract_text> ..