Michael G Kaplitt
Affiliation: Cornell University
- Subthalamic GAD gene therapy in a Parkinson's disease rat modelJia 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...
- Future and current surgical therapies in Parkinson's diseaseSimone 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...
- 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 lipofuscinosisDolan 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 31.7Â±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...
- Cholinergic interneurons in the nucleus accumbens regulate depression-like behaviorJennifer 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....
- Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerationsMark 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...
- Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological FunctionAnton 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...
- Improved sequence learning with subthalamic nucleus deep brain stimulation: evidence for treatment-specific network modulationHideo 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...
- Adeno-associated viral gene delivery in neurodegenerative diseasePeter 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...
- Reversal of depressed behaviors in mice by p11 gene therapy in the nucleus accumbensBrian 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...
- Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trialMichael 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...
- Novel mitochondrial substrates of omi indicate a new regulatory role in neurodegenerative disordersFelicity 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...
- Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNAStefan 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...
- AAV-mediated delivery of the caspase inhibitor XIAP protects against cisplatin ototoxicityLouis 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...
- PINK1 defect causes mitochondrial dysfunction, proteasomal deficit and alpha-synuclein aggregation in cell culture models of Parkinson's diseaseWencheng 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...
- Protection against cisplatin-induced ototoxicity by adeno-associated virus-mediated delivery of the X-linked inhibitor of apoptosis protein is not dependent on caspase inhibitionDylan 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...
- Inhibition of neuronal phenotype by PTEN in PC12 cellsSergei 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...
- Gene transfer and in vivo promoter analysis of the rat progesterone receptor using a herpes simplex virus viral vectorRoderick 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...
- Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disordersLisa 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...
- Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's diseaseAndrew 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...
- Network modulation in the treatment of Parkinson's diseaseKotaro 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...
- Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolismMarina 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...
- Neuroprotection via XIAP gene therapy in Huntington's diseaseMichael Kaplitt; Fiscal Year: 2007....
- PTEN Anti-Oncogene Influences on Neuronal Function & ToxicityMichael Kaplitt; Fiscal Year: 2007..abstract_text> ..
- XIAP Gene Therapy in Huntington's DiseaseMichael 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. ..