S Pablo Sardi

Summary

Affiliation: Genzyme Corporation
Country: USA

Publications

  1. pmc Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies
    S Pablo Sardi
    Genzyme, a Sanofi Company, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 110:3537-42. 2013
  2. doi request reprint Mutant GBA1 expression and synucleinopathy risk: first insights from cellular and mouse models
    S Pablo Sardi
    Genzyme, Sanofi Company, Framingham, Mass 01701, USA
    Neurodegener Dis 10:195-202. 2012
  3. pmc CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
    S Pablo Sardi
    Genzyme Corporation, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 108:12101-6. 2011
  4. pmc Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies
    Christopher M Treleaven
    Genzyme, a Sanofi Company, Framingham, Massachusetts 01701 9322, USA
    Mol Ther 20:1713-23. 2012
  5. pmc Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
    Marco A Passini
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA
    Sci Transl Med 3:72ra18. 2011
  6. pmc CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
    Marco A Passini
    Genzyme Corporation, 49 New York Avenue, Room 2410, Framingham, MA 01701, USA
    J Clin Invest 120:1253-64. 2010

Collaborators

Detail Information

Publications6

  1. pmc Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies
    S Pablo Sardi
    Genzyme, a Sanofi Company, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 110:3537-42. 2013
    ..Hence, increasing glucocerebrosidase activity in the CNS represents a potential therapeutic strategy for GBA1-related and non-GBA1-associated synucleinopathies, including PD...
  2. doi request reprint Mutant GBA1 expression and synucleinopathy risk: first insights from cellular and mouse models
    S Pablo Sardi
    Genzyme, Sanofi Company, Framingham, Mass 01701, USA
    Neurodegener Dis 10:195-202. 2012
    ..In summary, several leads connecting GBA1 mutations with α-synuclein misprocessing have emerged as potential targets for the treatment of GBA1-related synucleinopathies, and possibly, for non-GBA1-associated neurodegenerative diseases...
  3. pmc CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
    S Pablo Sardi
    Genzyme Corporation, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 108:12101-6. 2011
    ....
  4. pmc Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies
    Christopher M Treleaven
    Genzyme, a Sanofi Company, Framingham, Massachusetts 01701 9322, USA
    Mol Ther 20:1713-23. 2012
    ..These findings support the continued development of AAV-based therapies for the treatment of neurological disorders...
  5. pmc Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
    Marco A Passini
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA
    Sci Transl Med 3:72ra18. 2011
    ..These data demonstrate that central nervous system-directed ASO therapy is efficacious and that intrathecal infusion may represent a practical route for delivering this therapeutic in the clinic...
  6. pmc CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
    Marco A Passini
    Genzyme Corporation, 49 New York Avenue, Room 2410, Framingham, MA 01701, USA
    J Clin Invest 120:1253-64. 2010
    ..These data indicate that CNS-directed, AAV-mediated SMN augmentation is highly efficacious in addressing both neuronal and muscular pathologies in a severe mouse model of SMA...