Robin J Ziegler

Summary

Affiliation: Genzyme Corporation
Country: USA

Publications

  1. ncbi Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease
    Robin J Ziegler
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701 9322, USA
    Mol Genet Metab 97:35-42. 2009
  2. ncbi AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease
    Christine M Barbon
    Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA
    Mol Ther 12:431-40. 2005
  3. ncbi Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A
    Robin J Ziegler
    Genzyme Corporation, Framingham, Massachusetts, USA
    Mol Ther 15:492-500. 2007
  4. ncbi Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease
    Robin J Ziegler
    Genzyme Corporation, Framingham, MA 01701-9322, USA
    Hum Gene Ther 13:935-45. 2002
  5. ncbi Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice
    Joseph W Foley
    Genzyme Corporation, Framingham, Massachusetts, USA
    Mol Ther 18:1584-91. 2010
  6. ncbi Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression
    Jennifer B Nietupski
    Genzyme Corporation, Framingham, Massachusetts 01701, USA
    Mol Ther 19:1999-2011. 2011
  7. ncbi Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors
    Malgorzata Przybylska
    Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA
    J Gene Med 6:85-92. 2004
  8. ncbi AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice
    Robin J Ziegler
    Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA
    Mol Ther 9:231-40. 2004
  9. ncbi Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model
    John Marshall
    Genzyme Corporation, Framingham, Massachusetts, 01701 9322, USA
    Mol Ther 6:179-89. 2002
  10. ncbi Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse
    Marco A Passini
    Genzyme Corporation, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 104:9505-10. 2007

Collaborators

Detail Information

Publications15

  1. ncbi Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease
    Robin J Ziegler
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701 9322, USA
    Mol Genet Metab 97:35-42. 2009
    ..Hence, pulmonary administration of rhASM may represent an alternative route of delivery to address the visceral pathology associated with ASM deficiency...
  2. ncbi AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease
    Christine M Barbon
    Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA
    Mol Ther 12:431-40. 2005
    ..Together, these data support the continued development of AAV8-mediated hepatic gene transfer as an approach to treat the visceral manifestations observed in individuals with acid sphingomyelinase deficiency...
  3. ncbi Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A
    Robin J Ziegler
    Genzyme Corporation, Framingham, Massachusetts, USA
    Mol Ther 15:492-500. 2007
    ..Adoptive transfer of T cells isolated from the spleens of immunotolerized mice suppressed the formation of antibodies in naïve recipient animals, suggesting the possible role of regulatory T cells in effecting this state...
  4. ncbi Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease
    Robin J Ziegler
    Genzyme Corporation, Framingham, MA 01701-9322, USA
    Hum Gene Ther 13:935-45. 2002
    ..In this regard, we showed that pretreatment of mice with gamma globulins also resulted in significantly enhanced adenovirus-mediated transduction and expression of alpha-galactosidase A in the liver...
  5. ncbi Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice
    Joseph W Foley
    Genzyme Corporation, Framingham, Massachusetts, USA
    Mol Ther 18:1584-91. 2010
    ..These findings highlight the importance of treating Pompe skeletal muscle before pathology becomes irreversible, and suggest that adjunctive therapies may not be effective without first clearing skeletal muscle glycogen storage with GAA...
  6. ncbi Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression
    Jennifer B Nietupski
    Genzyme Corporation, Framingham, Massachusetts 01701, USA
    Mol Ther 19:1999-2011. 2011
    ..They also suggest some current limits on achieving clinically useful antibody reduction and therapeutic benefit for lysosomal storage diseases using a systemic AAV8-based approach...
  7. ncbi Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors
    Malgorzata Przybylska
    Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA
    J Gene Med 6:85-92. 2004
    ..However, substantial improvements in expression, especially in critical organs such as the kidney, are required before these vectors can become a viable approach to treat Fabry disease and other lysosomal storage disorders...
  8. ncbi AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice
    Robin J Ziegler
    Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA
    Mol Ther 9:231-40. 2004
    ..Together, these results demonstrate that AAV2-mediated gene transfer that limits the expression of alpha-galactosidase A to the liver may be a viable strategy for treating Fabry disease...
  9. ncbi Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model
    John Marshall
    Genzyme Corporation, Framingham, Massachusetts, 01701 9322, USA
    Mol Ther 6:179-89. 2002
    ..Together, these data demonstrate the potential for use of in vivo gene therapy vectors for treating Gaucher disease...
  10. ncbi Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse
    Marco A Passini
    Genzyme Corporation, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 104:9505-10. 2007
    ..These data demonstrate that combination therapy is a promising therapeutic modality for treating NPD and suggest a potential strategy for treating disease indications that cause both visceral and CNS pathologies...
  11. ncbi Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice
    Robin J Ziegler
    Genzyme, Framingham, MA 01701, USA
    Hum Gene Ther 19:609-21. 2008
    ..However, early therapeutic intervention is required to maintain significant muscle function and should be an important consideration in the management and treatment of Pompe disease...
  12. ncbi Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease
    Yunxiang Zhu
    Genzyme Corporation, Framingham, Massachusetts 01701 9322, USA
    Mol Ther 17:954-63. 2009
    ..These findings lend support to early therapeutic intervention with a targeted enzyme as important considerations in the management of Pompe disease...
  13. ncbi Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy
    Gregory D Hurlbut
    Genzyme Corporation, Framingham, Massachusetts, USA
    Mol Ther 18:1983-94. 2010
    ....
  14. ncbi Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion
    Robin J Ziegler
    Genzyme Corporation, Framingham, MA, USA
    Exp Neurol 231:261-71. 2011
    ..Additional optimization of an ICV delivery approach may provide a therapeutic option for LSDs with neurologic involvement...
  15. ncbi Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease
    Chester Li
    Genzyme Corporation, 31 New York Avenue, Framingham, Massachusetts 01701-9322, USA
    Mol Ther 5:745-54. 2002
    ..Together, these results suggest that the lung may be a viable alternate depot organ for the production and systemic secretion of alpha-galactosidase A for Fabry disease...