Michael G Kaplitt

Summary

Affiliation: Cornell University
Country: USA

Publications

  1. ncbi request reprint Subthalamic GAD gene therapy in a Parkinson's disease rat model
    Jia Luo
    Functional Genomics and Translational Neuroscience Laboratory, Department of Molecular Medicine and Pathology, University of Auckland, Auckland, New Zealand
    Science 298:425-9. 2002
  2. ncbi request reprint Future and current surgical therapies in Parkinson's disease
    Simone A Betchen
    Weill Medical College of Cornell University, New York, NY 10021, USA
    Curr Opin Neurol 16:487-93. 2003
  3. doi request reprint Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis
    Dolan Sondhi
    Department of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA
    Hum Gene Ther Methods 23:324-35. 2012
  4. pmc Cholinergic interneurons in the nucleus accumbens regulate depression-like behavior
    Jennifer L Warner-Schmidt
    Laboratory of Molecular and Cellular Neuroscience, The Rockefeller University, New York, NY 10065, USA
    Proc Natl Acad Sci U S A 109:11360-5. 2012
  5. pmc Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations
    Mark M Souweidane
    Department of Neurological Surgery, Weill Cornell Medical College, Cornell University, New York, New York 10021, USA
    J Neurosurg Pediatr 6:115-22. 2010
  6. pmc Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological Function
    Anton Orlin
    Department of Ophthalmology, Weill Cornell Medical College, New York, New York, United States of America
    PLoS ONE 8:e73128. 2013
  7. doi request reprint Improved sequence learning with subthalamic nucleus deep brain stimulation: evidence for treatment-specific network modulation
    Hideo Mure
    Center for Neurosciences, The Feinstein Institute for Medical Research, NY, USA
    J Neurosci 32:2804-13. 2012
  8. doi request reprint Adeno-associated viral gene delivery in neurodegenerative disease
    Peter F Morgenstern
    Laboratory of Molecular Neurosurgery, Department of Neurological Surgery, Weill Cornell Medical College, New York, NY, USA
    Methods Mol Biol 793:443-55. 2011
  9. pmc Reversal of depressed behaviors in mice by p11 gene therapy in the nucleus accumbens
    Brian Alexander
    Laboratory of Molecular Neurosurgery, Department of Neurological Surgery, Weill Cornell Medical College, New York, NY 10065, USA
    Sci Transl Med 2:54ra76. 2010
  10. ncbi request reprint Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
    Michael G Kaplitt
    Department of Neurological Surgery, Weill Medical College of Cornell University, New York, NY, USA
    Lancet 369:2097-105. 2007

Research Grants

Collaborators

  • Mark M Souweidane
  • Samuel H Selesnick
  • Donald J D'Amico
  • David Eidelberg
  • Carol A Tamminga
  • Per Svenningsson
  • Matthew J During
  • Patricia A Lawlor
  • Simone A Betchen
  • Andrew S Feigin
  • Marina Emborg
  • Yilong Ma
  • Ben Z Roitberg
  • DONALD WELLS PFAFF
  • M Carbon
  • Chenjian Li
  • Dolan Sondhi
  • Sergei Musatov
  • Ronald G Crystal
  • Neil R Hackett
  • Barry Kosofsky
  • Stefan Worgall
  • Dylan K Chan
  • Anton Orlin
  • Kaleb Yohay
  • Jennifer L Warner-Schmidt
  • Hideo Mure
  • Peter F Morgenstern
  • Paul Greengard
  • Margarita Arango-Lievano
  • Douglas Ballon
  • Brian Alexander
  • Felicity Johnson
  • Wencheng Liu
  • Stephen M Kaminsky
  • Vijay Dhawan
  • Jonathan P Dyke
  • Bruce M Greenwald
  • Louis B Cooper
  • Kotaro Asanuma
  • Lisa M Arkin
  • Roderick E M Scott
  • Jia Luo
  • Jason G Mezey
  • Szilard Kiss
  • Matthew T Witmer
  • Matthew M Wessel
  • Maria Felice Ghilardi
  • Jonathan Dyke
  • John J Marshall
  • Nathaniel Heintz
  • Chris C Tang
  • Miklos Argyelan
  • Keith Purpura
  • Amanda J Rubin
  • Stephen M Kaminksy
  • Sebastien Monette
  • Eric F Schmidt
  • Ines Ibanez-Tallon
  • Linda Johnson
  • Sergei A Musatov
  • Roberta Marongiu
  • Marc Flajolet
  • Mary Vernov
  • Margarita Arango-Llievano
  • Subroto Ghose
  • Mihaela Stavarache
  • Mihaela Stavarche
  • Jennifer Warner-Schmidt
  • Therese Eriksson
  • Mihaela A Stavarache
  • M Flint Beal
  • Jordi Magrane
  • Cristofol Vives-Bauza
  • Simon Chang
  • Giovanni Manfredi
  • Sebastian Shaffer
  • Yanping Li
  • Ai Yamamoto
  • Rebeca Acín-Peréz-
  • Xin Yun Huang
  • Yingcai Tan
  • Nurunisa Neyzi
  • Madhu Mazumdar
  • Linda Heier
  • Minal V Kekatpure
  • Paul Christos
  • David M Lieberman
  • Joshua A Goldfein
  • Justin F Fraser

Detail Information

Publications21

  1. ncbi request reprint Subthalamic GAD gene therapy in a Parkinson's disease rat model
    Jia Luo
    Functional Genomics and Translational Neuroscience Laboratory, Department of Molecular Medicine and Pathology, University of Auckland, Auckland, New Zealand
    Science 298:425-9. 2002
    ..This strategy suggests that there is plasticity between excitatory and inhibitory neurotransmission in the mammalian brain that could be exploited for therapeutic benefit...
  2. ncbi request reprint Future and current surgical therapies in Parkinson's disease
    Simone A Betchen
    Weill Medical College of Cornell University, New York, NY 10021, USA
    Curr Opin Neurol 16:487-93. 2003
    ..The present review discusses surgical therapies for the treatment of Parkinson's disease and the status of experimental strategies currently in preclinical and clinical testing...
  3. doi request reprint Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis
    Dolan Sondhi
    Department of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA
    Hum Gene Ther Methods 23:324-35. 2012
    ..TPP-I activity was >2 SD over background in 31.7±8.1% of brain at 90 days. These findings establish the dose and safety profile for human clinical studies for the treatment of LINCL with AAVrh.10hCLN2...
  4. pmc Cholinergic interneurons in the nucleus accumbens regulate depression-like behavior
    Jennifer L Warner-Schmidt
    Laboratory of Molecular and Cellular Neuroscience, The Rockefeller University, New York, NY 10065, USA
    Proc Natl Acad Sci U S A 109:11360-5. 2012
    ....
  5. pmc Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations
    Mark M Souweidane
    Department of Neurological Surgery, Weill Cornell Medical College, Cornell University, New York, New York 10021, USA
    J Neurosurg Pediatr 6:115-22. 2010
    ..The operative technique, radiographic changes, and surgical complications are presented...
  6. pmc Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological Function
    Anton Orlin
    Department of Ophthalmology, Weill Cornell Medical College, New York, New York, United States of America
    PLoS ONE 8:e73128. 2013
    ..The spectrum of ophthalmic manifestations of LINCL and the relationship with neurological function has not been previously described...
  7. doi request reprint Improved sequence learning with subthalamic nucleus deep brain stimulation: evidence for treatment-specific network modulation
    Hideo Mure
    Center for Neurosciences, The Feinstein Institute for Medical Research, NY, USA
    J Neurosci 32:2804-13. 2012
    ..Selective modulation of overactive SMA-STN projection pathways may underlie the improvement in learning found with stimulation...
  8. doi request reprint Adeno-associated viral gene delivery in neurodegenerative disease
    Peter F Morgenstern
    Laboratory of Molecular Neurosurgery, Department of Neurological Surgery, Weill Cornell Medical College, New York, NY, USA
    Methods Mol Biol 793:443-55. 2011
    ..Here, we describe an efficient and reliable method for the production and purification of AAV serotype 2 vectors for both in vitro and in vivo applications...
  9. pmc Reversal of depressed behaviors in mice by p11 gene therapy in the nucleus accumbens
    Brian Alexander
    Laboratory of Molecular Neurosurgery, Department of Neurological Surgery, Weill Cornell Medical College, New York, NY 10065, USA
    Sci Transl Med 2:54ra76. 2010
    ..Normalization of p11 expression within this brain region with AAV-mediated gene therapy may be of therapeutic value...
  10. ncbi request reprint Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
    Michael G Kaplitt
    Department of Neurological Surgery, Weill Medical College of Cornell University, New York, NY, USA
    Lancet 369:2097-105. 2007
    ..We aimed to measure the safety, tolerability, and potential efficacy of transfer of glutamic acid decarboxylase (GAD) gene with adeno-associated virus (AAV) into the subthalamic nucleus of patients with Parkinson's disease...
  11. pmc Novel mitochondrial substrates of omi indicate a new regulatory role in neurodegenerative disorders
    Felicity Johnson
    Department of Neurological Surgery, Cornell University, Weill Medical College, New York, New York, USA
    PLoS ONE 4:e7100. 2009
    ..However higher dose treatment caused increased Omi expression and decreased levels of pdhb and idh3a transcripts. This implicates mitochondrial OMI in a novel mechanism relating to metabolism...
  12. doi request reprint Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
    Stefan Worgall
    Department of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA
    Hum Gene Ther 19:463-74. 2008
    ..On this basis, we propose that additional studies to assess the safety and efficacy of AAV-mediated gene therapy for LINCL are warranted...
  13. ncbi request reprint AAV-mediated delivery of the caspase inhibitor XIAP protects against cisplatin ototoxicity
    Louis B Cooper
    Department of Neurological Surgery, Weill Medical College of Cornell University, New York, New York 10021, USA
    Otol Neurotol 27:484-90. 2006
    ..Delivery of the gene encoding X-linked inhibitor of apoptosis (XIAP) using an adeno-associated viral (AAV) vector can protect against cisplatin-mediated ototoxicity...
  14. pmc PINK1 defect causes mitochondrial dysfunction, proteasomal deficit and alpha-synuclein aggregation in cell culture models of Parkinson's disease
    Wencheng Liu
    Department of Neurology and Neurosciences, Weill Medical College of Cornell University, New York, New York, USA
    PLoS ONE 4:e4597. 2009
    ..Our results indicate that it will be important to delineate the relationship between mitochondrial functional deficits, proteasome dysfunction and alpha-synclein aggregation...
  15. ncbi request reprint Protection against cisplatin-induced ototoxicity by adeno-associated virus-mediated delivery of the X-linked inhibitor of apoptosis protein is not dependent on caspase inhibition
    Dylan K Chan
    Weill Medical College, Cornell University, New York, New York 10021, USA
    Otol Neurotol 28:417-25. 2007
    ..Gene therapy with an adeno-associated viral (AAV) vector encoding the X-linked inhibitor of apoptosis protein (XIAP) in an animal model of cisplatin-induced ototoxicity can elucidate apoptotic pathways in the inner ear...
  16. pmc Inhibition of neuronal phenotype by PTEN in PC12 cells
    Sergei Musatov
    Laboratory of Neurobiology and Behavior, The Rockefeller University, New York, NY 10021, USA
    Proc Natl Acad Sci U S A 101:3627-31. 2004
    ..Our findings may shed new light on the role of this tumor suppressor during normal brain development and suggest a previously uncharacterized mechanism of PTEN action in neuron-like cells...
  17. ncbi request reprint Gene transfer and in vivo promoter analysis of the rat progesterone receptor using a herpes simplex virus viral vector
    Roderick E M Scott
    Neurobiology and Behavior, Rockefeller University, 1230 York Avenue, New York, NY 10021, USA
    Brain Res Mol Brain Res 114:91-100. 2003
    ..These results demonstrate that the 2.1-kb PR promoter fragment contains the sequence information required for correct tissue and hormonal regulation of PR...
  18. ncbi request reprint Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders
    Lisa M Arkin
    Department of Genetic Medicine, Weill Medical College of Cornell University, New York, NY 10021, USA
    Hum Gene Ther 16:1028-36. 2005
    ..Our approach to these challenges should provide a useful paradigm for investigators initiating other genetic medicine- based studies to treat inevitably fatal diseases...
  19. pmc Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease
    Andrew Feigin
    Center for Neurosciences, The Feinstein Institute for Medical Research, North Shore Long Island Jewish Health System, 350 Community Drive, Manhasset, NY 11030, USA
    Proc Natl Acad Sci U S A 104:19559-64. 2007
    ..Network biomarkers may be used as physiological assays in early-phase trials of experimental therapies for PD and other neurodegenerative disease...
  20. ncbi request reprint Network modulation in the treatment of Parkinson's disease
    Kotaro Asanuma
    Center for Neurosciences, Feinstein Institute for Medical Research, North Shore Long Island Jewish Health System, Manhasset, NY 11030, USA
    Brain 129:2667-78. 2006
    ..The modulation of pathological brain networks is a critical feature of the treatment response in parkinsonism...
  21. ncbi request reprint Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism
    Marina E Emborg
    Wisconsin National Primate Research Center, University of Wisconsin, Madison, WI, USA
    J Cereb Blood Flow Metab 27:501-9. 2007
    ..The changes in motor cortical glucose utilization observed after gene therapy are consistent with the modulation of metabolic brain networks associated with this disorder...

Research Grants4

  1. Neuroprotection via XIAP gene therapy in Huntington's disease
    Michael Kaplitt; Fiscal Year: 2007
    ....
  2. PTEN Anti-Oncogene Influences on Neuronal Function & Toxicity
    Michael Kaplitt; Fiscal Year: 2007
    ..abstract_text> ..
  3. XIAP Gene Therapy in Huntington's Disease
    Michael G Kaplitt; Fiscal Year: 2010
    ..Given our promising preliminary data and recent use of gene therapy in human Parkinson's disease, this application may also facilitate development of XIAP gene therapy for human HD. ..