Marco A Passini

Summary

Affiliation: Genzyme Corporation
Country: USA

Publications

  1. ncbi request reprint Gene delivery to the mouse brain with adeno-associated virus
    Marco A Passini
    Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, and Division of Neurology, Children s Hospital of Philadelphia, Philadelphia, PA, USA
    Methods Mol Biol 246:225-36. 2004
  2. pmc Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease
    James C Dodge
    Genzyme Corporation, One Mountain Road, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 102:17822-7. 2005
  3. ncbi request reprint Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis
    Marco A Passini
    Neuroscience, Genzyme Corporation, Framingham, Massachusetts 01701, USA
    J Neurosci 26:1334-42. 2006
  4. pmc 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
  5. ncbi request reprint AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease
    Marco A Passini
    Neuroscience, Genzyme Corporation, One Mountain Road, Framingham, MA 01701, USA
    Mol Ther 11:754-62. 2005
  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
  7. doi request reprint Prospects for the gene therapy of spinal muscular atrophy
    Marco A Passini
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701 9322, USA
    Trends Mol Med 17:259-65. 2011
  8. 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
  9. doi request reprint Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease
    James C Dodge
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA
    Exp Neurol 215:349-57. 2009
  10. 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

Collaborators

Detail Information

Publications30

  1. ncbi request reprint Gene delivery to the mouse brain with adeno-associated virus
    Marco A Passini
    Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, and Division of Neurology, Children s Hospital of Philadelphia, Philadelphia, PA, USA
    Methods Mol Biol 246:225-36. 2004
    ..Although AAV2 vectors were shown to remain predominately at the injection site, one study demonstrated that the vector itself may undergo axonal transport in inter-regional systems (9)...
  2. pmc Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease
    James C Dodge
    Genzyme Corporation, One Mountain Road, Framingham, MA 01701, USA
    Proc Natl Acad Sci U S A 102:17822-7. 2005
    ..Our results support the continued development of AAV based vectors for gene therapy of the CNS manifestations in Niemann-Pick type A disease...
  3. ncbi request reprint Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis
    Marco A Passini
    Neuroscience, Genzyme Corporation, Framingham, Massachusetts 01701, USA
    J Neurosci 26:1334-42. 2006
    ..This study demonstrates that AAV-mediated TPP1 enzyme replacement corrects the hallmark cellular pathologies of cLINCL in the mouse model and raises the possibility of using AAV gene therapy to treat cLINCL patients...
  4. pmc 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...
  5. ncbi request reprint AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease
    Marco A Passini
    Neuroscience, Genzyme Corporation, One Mountain Road, Framingham, MA 01701, USA
    Mol Ther 11:754-62. 2005
    ..These findings show that the ASMKO brain is responsive to ASM replacement and that retrograde transport of AAV2 functions as a platform for widespread gene delivery and reversal of pathology in affected brain...
  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...
  7. doi request reprint Prospects for the gene therapy of spinal muscular atrophy
    Marco A Passini
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701 9322, USA
    Trends Mol Med 17:259-65. 2011
    ..Recent advances and findings from preclinical studies in animal models provide optimism that gene therapy might be an effective therapeutic strategy for treating SMA...
  8. 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...
  9. doi request reprint Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease
    James C Dodge
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA
    Exp Neurol 215:349-57. 2009
    ..These findings support the continued exploration of ICV delivery of recombinant lysosomal enzymes as a therapeutic modality for LSDs such as NPA that manifests substrate accumulation within the CNS...
  10. 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...
  11. 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...
  12. ncbi request reprint Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse
    Wendy W Yang
    Genzyme Corporation, One Mountain Road, Framingham, MA 01701 9322, USA
    Exp Neurol 207:258-66. 2007
    ..These results indicate that intraparenchymal injection of hASM is associated with minimal toxicity and can lead to regional reductions in storage pathology in the ASMKO mouse...
  13. pmc Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A
    Jie Bu
    Rare Diseases Division, Genzyme Corporation, Framingham, Massachusetts 01701 9322, USA
    Mol Ther 20:1893-901. 2012
    ..The information from this study might help design new AAV-mediated enzyme replacement protocols for NPA and other neuronopathic LSDs in future clinical trials...
  14. ncbi request reprint Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease
    Mario A Cabrera-Salazar
    Genzyme Corporation, Framingham, Massachusetts 01701, USA
    Mol Ther 15:1782-8. 2007
    ..These data illustrate the importance of early intervention for enhanced therapeutic benefit, which may provide guidance in designing novel treatment strategies for cLINCL patients...
  15. pmc Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity
    James C Dodge
    Genzyme Corporation, Framingham, Massachusetts 01701 9322, USA
    Mol Ther 16:1056-64. 2008
    ..Our findings highlight an innovative approach for delivering IGF-1 to the CNS...
  16. 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
    ....
  17. ncbi request reprint Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9-Survival Motor Neuron 1 for Spinal Muscular Atrophy
    Marco A Passini
    1 Rare Diseases Science, Genzyme, a Sanofi Company, Framingham, MA 01701
    Hum Gene Ther 25:619-30. 2014
    ..Thus, the extent of gene expression in motor neurons necessary to confer efficacy in SMA mice could be obtained in large-animal models, justifying the continual development of gene therapy for SMA. ..
  18. ncbi request reprint Identification of novel biomarkers for Niemann-Pick disease using gene expression analysis of acid sphingomyelinase knockout mice
    Rajwinder Dhami
    Department of Human Genetics, Mount Sinai School of Medicine, 1425 Madison Avenue, New York, NY 10029, USA
    Mol Ther 13:556-64. 2006
    ..These studies illustrate the value of gene expression analysis for the identification of biomarkers, and provide new insight into the pathobiology of NPD...
  19. ncbi request reprint Adeno-associated virus vector-mediated transduction in the cat brain
    Charles H Vite
    School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
    Gene Ther 10:1874-81. 2003
    ..AAV5 did not result in detectable transduction in the cat brain...
  20. ncbi request reprint A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration
    David E Sleat
    Center for Advanced Biotechnology and Medicine, University of Medicine and Dentistry of New Jersey, Piscataway, New Jersey 08854, USA
    J Neurosci 24:9117-26. 2004
    ..In addition, the CLN2-targeted mouse also represents a valuable model for the evaluation of different therapeutic strategies...
  21. ncbi request reprint Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream
    Marco A Passini
    Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
    J Neurosci 22:6437-46. 2002
    ..Gene transfer to specific neuronal circuits or cells in migratory pathways may facilitate delivery to the global brain lesions found in these disorders...
  22. pmc Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice
    Richard L Sidman
    Department of Neurology RLS, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA
    J Neuropathol Exp Neurol 67:803-18. 2008
    ..A better understanding of the basis for clinical manifestations is needed to correlate CNS pathology with Pompe disease manifestations...
  23. ncbi request reprint Effective gene therapy for an inherited CNS disease in a large animal model
    Charles H Vite
    School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA
    Ann Neurol 57:355-64. 2005
    ....
  24. doi request reprint Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis
    Michael Chang
    Department of Molecular Physiology and Biophysics, University of Iowa, Iowa City, Iowa 52242, USA
    Mol Ther 16:649-56. 2008
    ..This data demonstrates that intraventricular enzyme delivery to the CNS is feasible and may be of therapeutic value...
  25. ncbi request reprint AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy
    Mohammad A Rafi
    Department of Neurology, Jefferson Medical College, Philadelphia, PA 19107, USA
    Mol Ther 11:734-44. 2005
    ..Additional initiatives may be required to prevent the onset of disease and reverse the course of the disease in animal models and human patients...
  26. pmc Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mi
    Marco A Passini
    Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
    J Virol 77:7034-40. 2003
    ....
  27. ncbi request reprint Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible
    Gregory G Heuer
    Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
    Ann Neurol 52:762-70. 2002
    ..Treatment with a recombinant viral vector to correct the enzymatic defect quantitatively reversed the neurodegenerative lesions in targeted regions to normal levels...
  28. ncbi request reprint Genetically modified NT2N human neuronal cells mediate long-term gene expression as CNS grafts in vivo and improve functional cognitive outcome following experimental traumatic brain injury
    Deborah J Watson
    Department of Pathobiology, Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA
    J Neuropathol Exp Neurol 62:368-80. 2003
    ..These results demonstrate that defined populations of genetically modified human NT2N neurons are a practical and effective platform for stable ex vivo gene delivery into the CNS...
  29. ncbi request reprint Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors
    Deborah J Watson
    Department of Pathobiology and Walter Flato Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
    Hum Gene Ther 16:49-56. 2005
    ..This vector exhibited limited penetration from the ventricle to other structures, which was significantly different from the previously reported patterns of transduction after intraventricular injection of AAV1 and AAV2 vectors...
  30. ncbi request reprint Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins
    Deborah J Watson
    Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 5:528-37. 2002
    ....