Research Topics
Species | Marco A PassiniSummaryAffiliation: Genzyme Corporation Country: USA Publications
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Detail Information
Publications
Gene delivery to the mouse brain with adeno-associated virusMarco 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)...
Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A diseaseJames 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...- Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosisMarco 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... - Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophyMarco 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... 
Prospects for the gene therapy of spinal muscular atrophyMarco 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...- CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophyMarco 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... - Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouseMarco 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... - AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A diseaseMarco 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... - Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A diseaseJames 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... - Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathiesS Pablo Sardi
Genzyme, a Sanofi Company, Framingham, MA 01701
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... - Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodiesChristopher 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... - Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouseWendy 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... - Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type AJie 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... - Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten diseaseMario 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... - Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activityJames 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... - CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathyS Pablo Sardi
Genzyme Corporation, Framingham, MA 01701, USA
Proc Natl Acad Sci U S A 108:12101-6. 2011.... - Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease miceRichard 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... - 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 miMarco 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.... - Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory streamMarco 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... - Effective gene therapy for an inherited CNS disease in a large animal modelCharles H Vite
School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA
Ann Neurol 57:355-64. 2005.... - Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosisMichael 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... - AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophyMohammad 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... - Identification of novel biomarkers for Niemann-Pick disease using gene expression analysis of acid sphingomyelinase knockout miceRajwinder 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... - 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 neurodegenerationDavid 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... - Adeno-associated virus vector-mediated transduction in the cat brainCharles 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... - Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversibleGregory 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... - 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 injuryDeborah 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... - Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectorsDeborah 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... - Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteinsDeborah 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....