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Research Grants

Research Grants
Funded by the NNPDF and CCNNPDF


Note: The project status shown below reflects only the Foundation’s involvement. A number of the studies listed have been continued or expanded under the sponsorship of NIH and other organizations.

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Research Fellowships Funded by the NNPDF and CCNNPDF

Clinical Research and Services Funded by the NNPDF


GRANT #52
Melissa Wasserstein, MD
Mount Sinai School of Medicine, New York

Niemann-Pick Disease: Genotype/Phenotype Analyses and Molecular Based Therapy


GRANT #51
Charles Vite, DVM, PhD
School of Veterinary Medicine, University of Pennsylvania – Philadelphia

NPC Cat Colony “Fellowships of the Cats II”


GRANT #50
Charles Vite, DVM, PhD
School of Veterinary Medicine, University of Pennsylvania – Philadelphia

NPC Cat Colony “Fellowships of the Cats I”


GRANT #49
Jackson Laboratory Repository

Processing the NPD Mouse Model Developed by Dr. Richard Pagano of the Mayo Clinic College of Medicine


GRANT #48
Edward H. Schuchman, PhD
Mount Sinai School of Medicine, New York, NY (1 Year)
Construction & Characterization of New Mouse Models for Types A & B Niemann-Pick


GRANT #47
Frances M. Platt, PhD & Emyr Lloyd-Evans, PhD
University of Oxford, Oxford, UK (2 years)
Targeting Sphingosine Storage as a New Therapeutic approach for Niemann-Pick Type C


GRANT #46
Lysosomal Storage Disease Research Consortium (Multiple) (1 year)
Additional Contribution to Lysosomal Storage Disease (LSD) Research Consortium for Research Into Issues of Common Interest to all LSD Support Groups


GRANT #45
Frances M. Platt, PhD
University of Oxford (1 year)
Evaluation of Anti-Inflammatory Intervention (NSAIDS) as an Adjunctive Therapy in Niemann-Pick Type C1 Disease


GRANT #44
Lysosomal Storage Disease Research Consortium (Multiple)
Additional Contribution to LSD Research Consortium for Research Into Issues of Common Interest to all LSD Support Groups


GRANT #43
Maria D. Ledesma, PhD
Catholic University of Leuven, Leuven, Belgium (1 year)
Analysis of Dendritic Spine Alterations in Niemann-Pick Disease Type A Neurons


GRANT #42
Kostantin Dobrenis, PhD
Albert Einstein College of Medicine of Yeshiva University, Bronx, NY  USA  (18 Months)
Establishment of Central Nervous System Cell Lines from NPC-1 Disease Mice for Pathogenic and Therapeutic Studies


GRANT #41
Frances M. Platt, PhD
University of Oxford, Oxford, England, UK
Therapeutic Monitoring of Glycolipid Storage Diseases in the Clinic Using Cell Biological Assays and Proteomics


GRANT #40
Lysosomal Storage Disease Research Consortium (Multiple)
Lysosomal Storage Disease Research Consortium Contribution for NNPDF to Join Consortium


GRANT #39
Robert A. Maue, PhD
Dartmouth Medical School, Hanover, NH, USA
Maintenance of Colony of NPC1 Mice with GFP-Tagged Purkinje Neurons


GRANT #38
Thomas J. Kelley, PhD
Reserve University, Cleveland, OH, USA

NOS2 Regulation in NPC


GRANT #37
Richard E. Pagano, PhD and David L. Marks, PhD
Mayo Clinic and Foundation, Rochester, MN, USA

Reduction of Stored Lipids in Niemann-Pick Type C Mice by RAB Protein Expression


GRANT #36
Maria D. Ledesma, PhD
Fundazione Cavalieri Ottolenghi, Turin, Italy (1 Year)
Organization of Synapses in Niemann-Pick Disease Type A


GRANT #35
Synthia Mellon, PhD 
University of California, San Francisco, San Francisco, CA, USA (1 year)
SSRIs as a Potential Therapy for NPC


GRANT #34
Stuart A. Lipton, MD, PhD
Burnham Institute, La Jolla, CA, USA (1 Year)
NMDA Receptor Hypofunction in Niemann-Pick Disease Type A


GRANT #33
Makoto Michikawa, MD, PhD 
National Institute for Longevity Sciences, Aichi, Japan (1 Year)
Cellular and Molecular Mechanisms Underlying Neurodegeneration in Niemann- Pick Type C Disease


GRANT #32
Timothy M. Cox, MD, FRCP, FMedSci &
Robin Henry Lachmann, MA, MB, Bchir, PhD 

University of Cambridge, Cambridge, UK (10 Months)
Therapeutic Gene Delivery to the Cerebellum in Niemann-Pick Type C


GRANT #31
Maria D. Ledesma, PhD
Fundazione Cavalieri Ottolenghi, Turin, Italy (1 year)
Niemann-Pick Disease Type A: Molecular Analysis of Neuronal Membrane Maturation and Synapse Formation


GRANT #30
Anita H. Corbett, PhD
Emory University School of Medicine, Atlanta, GA USA (1 year)
Functional Analysis of NPC1 


GRANT #29
Melanie Jean Dobson, PhD
Dalhousie University, Halifax, Nova Scotia, CAN (1 year)
A Yeast System for Analysis of Sterol Trafficking Defects in NPC


GRANT #28
Daniel S. Ory, MD 
Washington University School of Medicine, St. Louis, MO
The Role of HE1 in Cholesterol Trafficking


GRANT #27
Skadrite Krisans, PhD 
San Diego State University, San Diego, CA (1 year)
Role of Peroxisomal Cholesterol/Myelin Formation in the CNS Pathology of the NPC Mouse


GRANT #26
Anthony H. Futerman, PhD 
Weizmann Institute, Rehovot, Israel (1 year)
Neuronal Development & Death in Type A Niemann-Pick Disease


GRANT #25
Mary Anna Thrall, DVM
Colorado State University; Fort Collins, CO (1 year)
Characterization of Feline Niemann-Pick Type C


GRANT #24
Yiannis A. Ioannou, PhD 
Mount Sinai School of Medicine, New York, NY (1 year)
Functional Characterization of NPC1L1, a Human Homologue of NPC1


GRANT #23
Robert A. Maue, PhD
Dartmouth Medical School, Hanover, NH, USA (3 years)
Analysis of Growth Factor Actions and Membrane Polarity in Neurons from NP-C Mice


GRANT #22
Daniel S. Ory, MD
Washington University School of Medicine, St. Louis, MO
Molecular Cloning of the Niemann-Pick Type C2 Gene


GRANT #21
Cynthia J. Forehand, PhD 
University of Vermont, Burlington, VT
Glutamate Receptors and Neuronal Degeneration in Niemann-Pick Type C Disease


GRANT #20
Lisa D. Griffin, MD, PhD
University of California San Francisco, San Francisco, CA
Altered Neurosteroidogenesis in Niemann-Pick Type C Disease


GRANT #19
James Shayman, MD
University of Michigan, Ann Arbor, MI
Treatment of Niemann-Pick Type C Disease with Potent Inhibitors of Cerebroside Synthase


GRANT #18
Leslie P. Henderson, PhD
Dartmouth College, Hanover, NH
Analysis of NPC1 mRNA Expression in the Mouse Brain


GRANT #17
Daniel Ory, M.D.
Washington University School of Medicine, St. Louis, MO

Molecular Cloning of NPC2 Gene


GRANT #16
Lisa D. Griffin, MD, PhD
University of California San Francisco, San Francisco, CA
The Role of Neurosteroids in the Development of Niemann-Pick Type C


GRANT #15
Frederick R. Maxfield, PhD
Weill Medical College of Cornell University, New York, NY
Intracellular Cholesterol Trafficking in Niemann-Pick Type C Disease


GRANT #14
Jerry Reagan, PhD
Wake Forest University School of Medicine, Winston-Salem, NC
Acid Sphingomyelinase Regulates Cholesterol Egress from Lysosomes by an Alternative Pathway


GRANT #13
Daniel Ory, MD
Washington University School of Medicine, St. Louis, MO

Molecular Cloning of NPC2 Gene


GRANT #12
Jill Morris, PhD
National Institutes of Health, Bethesda, MD
Identification of Additional NPC1 Mutations


GRANT #11
Ira Tabas, MD, PhD
Columbia University, New York, NY
Phospholipid Metabolism and Cholesterol-Induced Cytotoxicity in Niemann-Pick C Macrophages


GRANT #10
Leslie P. Henderson, PhD
Dartmouth College, Hanover, NH
Analysis of NPC1 mRNA Expression in the Mouse Brain


GRANT #9
Marc Patterson, MD
Mayo Clinic, Rochester, MN
Lipid Trafficking in the Neurons of NPC Mice


GRANT #8
Synthia Mellon, PhD
University of California San Francisco, San Francisco, CA
Potential Involvement of Neurosteroids in the Neurobiological Dysfunctions of NPC


GRANT #7
Marc Patterson, MD
Mayo Clinic, Rochester, MN
Establish an International Registry (database) of NPC Families


GRANT #6
G. S. Tint, PhD
Veterans Administration Medical Center, East Orange, NJ
Accumulation of Oxidized Cholesterol as a Possible Cause of Pathologies Associated with Niemann-Pick Type C Disease


GRANT #5
John R. Sargent, PhD
University of Stirling, Stirling, Scotland, United Kingdom
Influence of Exogenous Fatty Acids on Levels of Cholesterol in Cultured Skin Fibroblasts from NPC Patients


GRANT #4
Wenda L. Greer, PhD
Dalhousie University, Halifax, Nova Scotia, Canada
Identification of Niemann-Pick Type D Patients with Arcadian Ancetsry as a Step Toward Isolation of the Defective Gene


GRANT #3
Charles C. Schwartz, MD
Medical College of Virginia, Richmond, VA
Quantitation of Cholesterol Transport and Tissue Cholesterol Pool Sizes In Vivo Niemann-Pick Type C Disease


GRANT #2
Laura Liscum, PhD
Tufts University School of Medicine, Boston, MA
Complementation Analyses of Niemann-Pick Type C Fibroblasts’ Cholesterol Transport in Defective CHO Cells


GRANT #1
Eugene Carstea, PhD
National Institutes of Health, Bethesda, MD (5 years)
Identify the Gene Responsible for Niemann-Pick Type C Disease


12/14/2016 jjb