Lamya S Shihabuddin

Summary

Affiliation: Genzyme Corporation
Country: USA

Publications

  1. ncbi request reprint Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology
    L S Shihabuddin
    Genzyme Corporation, Framingham, Massachusetts 01701, USA
    J Neurosci 24:10642-51. 2004
  2. pmc Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases
    Lamya S Shihabuddin
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701 9322, USA
    Neurotherapeutics 8:659-67. 2011
  3. doi request reprint Stem cell transplantation for neurometabolic and neurodegenerative diseases
    Lamya S Shihabuddin
    Genzyme Corporation, Science Center, Framingham, MA 01701, USA
    Neuropharmacology 58:845-54. 2010
  4. 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
  5. doi request reprint 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
  6. 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
  7. 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
  8. 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
  9. pmc Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis
    James C Dodge
    Genzyme, Framingham, MA 01701 9322, USA
    Proc Natl Acad Sci U S A 110:10812-7. 2013
  10. 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

Collaborators

Detail Information

Publications26

  1. ncbi request reprint Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology
    L S Shihabuddin
    Genzyme Corporation, Framingham, Massachusetts 01701, USA
    J Neurosci 24:10642-51. 2004
    ..Potentially, NPCs may serve as a useful gene transfer vehicle for the treatment of CNS pathology in other lysosomal storage diseases and neurodegenerative disorders...
  2. pmc Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases
    Lamya S Shihabuddin
    Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701 9322, USA
    Neurotherapeutics 8:659-67. 2011
    ..This review discusses some of the well-characterized neural stem cell types and their possible use in treating neuropathic lysosomal storage diseases such as the Niemann Pick A disease...
  3. doi request reprint Stem cell transplantation for neurometabolic and neurodegenerative diseases
    Lamya S Shihabuddin
    Genzyme Corporation, Science Center, Framingham, MA 01701, USA
    Neuropharmacology 58:845-54. 2010
    ....
  4. 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...
  5. doi request reprint 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...
  6. 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...
  7. 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...
  8. 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...
  9. pmc Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis
    James C Dodge
    Genzyme, Framingham, MA 01701 9322, USA
    Proc Natl Acad Sci U S A 110:10812-7. 2013
    ..Collectively, these data provide insights into the pathogenesis of ALS as well as potential targets for drug development. ..
  10. 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...
  11. 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...
  12. 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...
  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 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...
  15. 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...
  16. pmc Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone
    Jonathan A Fidler
    Genzyme Corporation, 49 New York Ave, Framingham, MA 01701 9322, USA
    FASEB J 25:4369-77. 2011
    ..680; females: r(2)=0.552) in ALS mice. These results suggest that stress is capable of accelerating disease progression and that strategies that modulate glucocorticoid metabolism might be a viable treatment approach for ALS...
  17. pmc AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice
    James C Dodge
    Genzyme Corporation, Framingham, Massachusetts 01701 9322, USA
    Mol Ther 18:2075-84. 2010
    ..These findings support the continued investigation of trophic factor-based therapies that target the CNS as a potential treatment of ALS...
  18. 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...
  19. 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
    ....
  20. pmc Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery
    Christopher M Treleaven
    Genzyme Corporation, Framingham, Massachusetts, United States of America
    PLoS ONE 6:e16313. 2011
    ..Collectively, these data suggest that the efficacy observed following ICV delivery of hASM in ASMKO mice could be scaled to CNS of the rat...
  21. doi request reprint Relationship between neuropathology and disease progression in the SOD1(G93A) ALS mouse
    Wendy W Yang
    Genzyme Corporation, 49 New York Ave, Framingham, MA 01701 9322, USA
    Exp Neurol 227:287-95. 2011
    ..Hence, our findings support a role for astrocytes in modulating the progression of non-cell autonomous degeneration of motor neurons, with microglia playing a role in clearing degenerating neurons...
  22. 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...
  23. 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...
  24. ncbi request reprint Optimized preservation of CNS morphology for the identification of glycogen in the Pompe mouse model
    Tatyana V Taksir
    Department of Pathology, Genzyme Corporation, One Mountain Road, Framingham, MA 01701 9322, USA
    J Histochem Cytochem 55:991-8. 2007
    ..The modified paraffin method with periodic acid postfixation resulted in improved tissue morphology and glycogen preservation. Both techniques provide accurate anatomic evaluation of the glycogen distribution in Pompe mouse brain...
  25. 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...
  26. pmc Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease
    Lisa M Stanek
    Genzyme a Sanofi Company, Framingham, MA 01701
    Hum Gene Ther 25:461-74. 2014
    ..Collectively, these results support the continued development of AAV-mediated RNAi as a therapeutic strategy for HD...