Paola Leone

Summary

Affiliation: New Jersey
Country: USA

Publications

  1. pmc Long-term follow-up after gene therapy for canavan disease
    Paola 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
  2. pmc Aspartoacylase supports oxidative energy metabolism during myelination
    Jeremy 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
  3. ncbi request reprint Dietary triheptanoin rescues oligodendrocyte loss, dysmyelination and motor function in the nur7 mouse model of Canavan disease
    Jeremy 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
  4. ncbi request reprint Serial 1H-MRS in GM2 gangliosidoses
    Mitra 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
  5. doi request reprint Endogenous aspartoacylase expression is responsive to glutamatergic activity in vitro and in vivo
    Jeremy S Francis
    Cell and Gene Therapy Center, UMDNJ School of Osteopathic Medicine, Stratford, New Jersey, USA
    Glia 59:1435-46. 2011

Detail Information

Publications5

  1. pmc Long-term follow-up after gene therapy for canavan disease
    Paola 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...
  2. pmc Aspartoacylase supports oxidative energy metabolism during myelination
    Jeremy 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...
  3. ncbi request reprint Dietary triheptanoin rescues oligodendrocyte loss, dysmyelination and motor function in the nur7 mouse model of Canavan disease
    Jeremy 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. ..
  4. ncbi request reprint Serial 1H-MRS in GM2 gangliosidoses
    Mitra 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...
  5. doi request reprint Endogenous aspartoacylase expression is responsive to glutamatergic activity in vitro and in vivo
    Jeremy S Francis
    Cell and Gene Therapy Center, UMDNJ School of Osteopathic Medicine, Stratford, New Jersey, USA
    Glia 59:1435-46. 2011
    ....

Research Grants5

  1. Aspartoacylase gene transfer in brain/Canavan Disease
    Paola Leone; Fiscal Year: 2003
    ..abstract_text> ..
  2. Aspartoacylase gene transfer in brain/Canavan Disease
    Paola Leone; Fiscal Year: 2002
    ..abstract_text> ..
  3. Aspartoacylase gene transfer in brain/Canavan Disease
    Paola Leone; Fiscal Year: 2004
    ..abstract_text> ..
  4. Aspartoacylase gene transfer in brain/Canavan Disease
    Paola Leone; Fiscal Year: 2004
    ..abstract_text> ..