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Research Grants Jointly Sponsored 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.

(To see a listing of the Fellowships jointly sponsored by the NNPDF and CCNNPDF, click here.)

(To see a listing of the Clinical Research and Services funded by the NNPDF, click here.)

Item # Date Researcher Project Additional Information
52 10/2014
– 10/2016
Wasserstein
Melissa Wasserstein, M.D.
Mount Sinai School of MedicineNew York
“Niemann-Pick Disease: Genotype/Phenotype Analyses and Molecular Based Therapy”

$100,000
Jointly sponsored by the NNPDF & CCNNPDF

In Progress

October 2015  Project Summary

April 2015 Project Summary

October 2014 Project Summary

51 10/2013
– 12/2015
vite7995
Charles Vite, D.V.M., Ph.D.
School of Veterinary Medicine
University of Pennsylvania – Philadelphia
NPC Cat colony “Fellowships of the Cats II”

$100,000
Jointly sponsored by NNPDF and CCNNPDF

Extended additional two months

October 2015 Progress Report

April 2015 Project Summary

October 2014 Progress Report

April 2014 Progress Report

October 2013 Project Summary

2013 Family Conference Presentation
(requires Powerpoint)

2013 Family Conference Presentation
(PDF)

 

50 08/2011
– 09/2013
vite7995
Charles Vite, D.V.M., Ph.D.
School of Veterinary Medicine
University of Pennsylvania – Philadelphia
NPC Cat colony “Fellowships of the Cats I”

$100,000
Jointly sponsored by NNPDF and CCNNPDF

Completed

Faculty page

September 2013 Final Report

April 2013 Progress Summary

2012 Cyclodextrin Report

August 2012 Progress Summary

49 8/2009 Jackson Laboratory Repository Processing the NPD mouse model developed by Dr. Richard Pagano of the Mayo Clinic College of Medicine

$2500
Jointly sponsored by NNPDF and CCNNPDF

Completed

48 4/1/2008 – 3/31/2009 1097530_000
Edward H. Schuchman, Ph.D.
(Mount Sinai School of Medicine, New York,NY, USA)
(1 Year)
Construction & Characterization of New Mouse Models for Types A & B Niemann-Pick

$46,593
Jointly sponsored by NNPDF and CCNNPDF

Completed

More Details…

Sept 2008 Progress Summary

May 2009 Progress Summary

47 3/1/2008 – 2/28/2010 FranPlattphoto_001
Frances M. Platt, Ph.D. & Emyr Lloyd-Evans, Ph.D.
(University of Oxford, Oxford, UK)
(2 years)
Targeting sphingosine storage as a new therapeutic approach for Niemann-Pick type C

$161,097
Jointly sponsored by NNPDF and CCNNPDF

Completed

More Details…

Oct 2008 Progress Summary

Dec 2010 Progress Summary

46 4/1/2007 – 3/31/2008 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
(See also Grant # 40)
$10,000
Jointly sponsored by NNPDF and CCNNPDFCompleted
More Details…
45 3/1/2007 – 2/29/2008 FranPlattphoto_001
Frances M. Platt, Ph.D.
(University of Oxford)
(1 year)
Evaluation of anti-inflammatory intervention (NSAIDS) as an adjunctive therapy in Niemann-Pick Type C1 disease.
$99,090
Jointly sponsored by NNPDF and CCNNPDFCompleted
More Details…

 

44 4/1/2006 – 3/31/2007 Lysosomal Storage Disease Research Consortium (Multiple)
(1 year)
Additional contribution to LSD Research Consortium for research into issues of common interest to all LSD support groups
(See also Grant # 40)
$10,000Completed
More Details…
43 9/1/2006 – 8/31/2007 MariaLedesma
Maria D. Ledesma, Ph.D.
(Catholic University of Leuven; Leuven; Belgium)
(1 year)
Analysis of Dendritic Spine Alterations in Niemann-Pick Disease Type A Neurons
$19,760
Bridge FundingCompleted
More Details…

2014 ASMD Research Publication

 

42 6/1/2006 – 12/31/2007 Kostantin Dobrenis, Ph.D.
(Albert Einstein College of Medicine of Yeshiva University; Bronx, New York; USA)
(18 months)
Establishment of Central Nervous System Cell Lines from NPC-1 Disease Mice for Pathogenic and Therapeutic Studies
$80,240Completed
More Details…
41 5/1/2006 – 11/30/2006 FranPlattphoto_001
Frances M. Platt, Ph.D.
(University of Oxford; Oxford, England; UK)
(7 months)
Therapeutic Monitoring of Glycolipid Storage Diseases in the Clinic Using Cell Biological Assays and Proteomics
$50,000
Bridge FundingCompleted
More Details…
40 4/1/2004 – 3/31/2006 Lysosomal Storage Disease Research Consortium (Multiple) Lysosomal Storage Disease Research Consortium Contribution for NNPDF to Join Consortium
$10,000Completed
More Details…
39 4/15/2005 – 4/14/2006 Robert A. Maue, Ph.D.
(Dartmouth Medical School; Hanover, New Hampshire; USA)
(1 year)
Maintenance of Colony of NPC1 mice with GFP-tagged Purkinje neurons
$6,000
Bridge FundingCompleted
More Details…
38 5/1/2005 – 4/30/2006 Thomas J. Kelley, Ph.D.
(Case Western Reserve University; Cleveland, Ohio; USA)
(1 year)
NOS2 regulation in NPC
$55,000Major funding provided by
the Niemann-Pick Disease Group (UK).Completed
More Details…
37 4/1/2005 – 4/1/2007 Richard E. Pagano, Ph.D. and David L. Marks, Ph.D.
(Mayo Clinic and Foundation; Rochester, Minnesota; USA)
(2 years)
Reduction of stored lipids in Niemann-Pick Type C mice by rab protein expression
$177,562Completed
More Details…
36 9/15/2004 – 9/14/2005 MariaLedesma
Maria D. Ledesma, Ph.D.
(Fundazione Cavalieri Ottolenghi; Turin, Italy)
(1 year)
Organization of Synapses in Niemann-Pick Disease Type A
$50,000Completed
More Details…
35 4/1/2004 – 3/31/2005 Synthia Mellon, Ph.D.
(University of California, San Franciso; San Francisco, California, USA)
(1 year)
SSRIs as a potential therapy for NP-C
$50,000Jointly funded with Dana’s Angels Research Trust . Total project cost is: $97,769.Completed
More Details…
34 3/1/2004 – 2/28/2005 Stuart A. Lipton, M.D., Ph.D.
(The Burnham Institute; La Jolla, California, USA)
(1 year)
NMDA receptor hypofunction in Niemann-Pick disease Type A
$50,000Completed
More Details…
33 9/15/2003 – 9/14/2004 Makoto Michikawa, M.D., Ph.D.
(National Institute for Longevity Sciences; Aichi, Japan)
(1 year)
Cellular and Molecular Mechanisms Underlying Neurodegeneration in Niemann- Pick Type C disease
$50,000Completed
More Details…
32 7/1/2003 – 8/31/2004 Timothy M. Cox, MD, FRCP, FMedSci & Robin Henry Lachmann, M.A., M.B., Bchir, Ph.D.
(University of Cambridge; Cambridge, United Kingdom)
(10 months)
Therapeutic Gene Delivery to the Cerebellum in Niemann-Pick Type C
$40,000
(Transitional Funding – used for PostDoc)Completed
More Details…
31 8/15/2002 – 8/14/2003 MariaLedesma
Maria D. Ledesma, Ph.D.
(Fundazione Cavalieri Ottolenghi; Turin, Italy)
(1 year)
Niemann-Pick Disease Type A: Molecular Analysis of Neuronal Membrane Maturation and Synapse Formation
$50,000Completed
More Details…
30 8/1/2002 – 7/31/2003 Anita H. Corbett, Ph.D.
(Emory University School of Medicine; Atlanta, Georgia, USA)
(1 year)
Functional Analysis of NPC1
$50,000Completed
More Details…
29 2/15/2002 – 2/14/2003 Melanie Jean Dobson, Ph.D.
(Dalhousie University; Halifax, Nova Scotia, Canada)
(1 year)
A Yeast System for Analysis of Sterol Trafficking Defects in NPC
$42,000Completed
More Details…
28 8/15/2001 – 8/14/2003 DanOryFeb2009cropped004_001
Daniel S. Ory, M.D.
(Washington University School of Medicine; St. Louis, Missouri, USA)
(2 years)
The Role of HE1 in Cholesterol Trafficking
$154,147Completed
More Details…
27 8/15/2001 – 8/14/2002 Skadrite Krisans, Ph.D.
(San Diego State University; San Diego, California, USA)
(1 year)
Role of Peroxisomal Cholesterol/Myelin Formation in the CNS Pathology of the NPC Mouse
$50,000Completed
26 2/1/2001 – 1/31/2002 Anthony H. Futerman, Ph.D.
(Weizmann Institute; Rehovot, Israel)
(1 year)
Neuronal Development & Death in Type A Niemann-Pick Disease
$50,000Completed
More Details…
25 8/15/2000 – 8/14/2001 Mary Anna Thrall; DVM
(Colorado State University; Fort Collins, Colorado, USA)
(1 year)
Characterization of Feline Niemann-Pick Type C
$40,216Completed
Faculty page
24 9/1/2000 – 8/31/2001 YiannisIoannou1247510_001
Yiannis A. Ioannou, Ph.D.
(Mount Sinai School of Medicine; New York, New York, USA)
(1 year)
Functional Characterization of NPC1L1, a Human Homologue of NPC1
$60,000Completed
23 8/15/2000 – 8/14/2003 Robert A. Maue, Ph.D.
(Dartmouth College)
(3 years)
Analysis of Growth Factor Actions and Membrane Polarity in Neurons from NP-C Mice
$131,988
(No Cost Extension Received in August, 2002; Completion Date Changed to August, 2003)Completed
More Details…
22 April, 2000 DanOryFeb2009cropped004_001
Daniel S. Ory, M.D.
(Washington University School of Medicine; St. Louis, Missouri, USA)
Molecular Cloning of the Niemann-Pick Type C2 Gene
$125,752Completed
21 February, 2000 Cynthia J. Forehand, Ph.D.
(University of Vermont; Burlington, Vermont, USA)
Glutamate Receptors and Neuronal Degeneration in Niemann-Pick Type C Disease
$27,107Completed
20 April, 2000 Lisa D. Griffin, M.D., Ph.D.
(University of California – San Francisco; San Francisco, California, USA)
Altered Neurosteroidogenesis in Niemann-Pick Type C Disease
$40,000
(Extension of Grant # 16)Completed
19 February, 2000 James Shayman, M.D.
(University of Michigan; Ann Arbor, Michigan, USA)
Treatment of Niemann-Pick Type C Disease with Potent Inhibitors of Cerebroside Synthase
$40,000Completed
18 August, 1999 Leslie P. Henderson
(Dartmouth College, Hanover, New Hampshire, USA)
Analysis of NPC1 mRNA Expression in the Mouse Brain
$14,592
(Extension of Grant # 10)Completed
17 July, 1999 DanOryFeb2009cropped004_001
Daniel Ory, M.D.
(Washington University School of Medicine; St. Louis, Missouri, USA)
Molecular Cloning of NPC2 Gene
$110,000
Extended (See Grant # 22)
16 June, 1999 Lisa Griffin, M.D., Ph.D.
(University of California – San Francisco; San Francisco, California, USA)
The Role of Neurosteroids in the Development of Niemann-Pick Type C
$34,000Completed
15 June, 1999 Frederick R. Maxfield, Ph.D.
(Weill Medical College of Cornell University; New York, New York, USA)
Intracellular Cholesterol Trafficking in Niemann-Pick Type C Disease
$15,000Completed
14 September, 1998 Jerry Reagan, Ph.D.
(Wake Forest University School of Medicine; Winston-Salem, North Carolina, USA)
Acid Sphingomyelinase Regulates Cholesterol Egress from Lysosomes by an Alternative Pathway
$15,000Completed
13 April, 1998 DanOryFeb2009cropped004_001
Daniel Ory, M.D.
(Washington University School of Medicine; St. Louis, Missouri, USA)
Molecular Cloning of NPC2 Gene
$60,000
Extended (See Grant #17)
12 February, 1998 Jill Morris, Ph.D.
(National Institutes of Health; Bethesda, Maryland, USA)
Identification of Additional NPC1 Mutations
$31,000Completed
11 March, 1998 Ira Tabas, M.D., Ph.D.
(Columbia University; New York, New York, USA)
Phospholipid Metabolism and Cholesterol-Induced Cytotoxicity in Niemann-Pick C Macrophages
$15,000Completed
10 March, 1998 Leslie P. Henderson, Ph.D.
(Dartmouth College, Hanover, New Hampshire, USA)
Analysis of NPC1 mRNA Expression in the Mouse Brain
$14,742
Extended (See Grant # 18)
9 March, 1997 MarcPatterson10847111
Marc Patterson, M.D.
(Mayo Clinic; Rochester, Minnesota, USA)
Lipid Trafficking in the Neurons of NPC Mice
$11,000Completed
8 January, 1997 Synthia Mellon, Ph.D.
(University of California – San Francisco; San Francisco, California; USA)
Potential Involvement of Neurosteroids in the Neurobiological Dysfunctions of NP-C
$30,044Completed
7 October, 1996 MarcPatterson10847111
Marc Patterson, M.D.
(Mayo Clinic; Rochester, Minnesota, USA)
Establish an International Registry (database) of NPC Families
$10,213
6 July, 1996 G. S. Tint, Ph.D.
(Veterans Adminstration Medical Center; East Orange, New Jersey, USA)
Accumulation of Oxidized Cholesterol as a Possible Cause of Pathologies Associated with Niemann-Pick Type C Disease
$14,000Completed
5 June, 1996 John R. Sargent, Ph.D.
(University of Stirling; Stirling, Scotland, United Kingdom)
Influence of Exogenous Fatty Acids on Levels of Cholesterol in Cultured Skin Fibroblasts from NPC Patients
$28,050Completed
4 January, 1996 Wenda L. Greer, Ph.D.
(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
$12,357Completed
3 Nov, 1995 Charles C. Schwartz, M.D.
(Medical College of Virginia; Richmond, Virginia, USA)
Quantitation of Cholesterol Transport and Tissue Cholesterol Pool Sizes In Vivo Niemann-Pick Type C Disease
$32,000Completed
2 April, 1994 Laura Liscum, Ph.D.
(Tufts University School of Medicine; Boston, Massachusetts, USA)
Complementation Analyses of Niemann-Pick Type C Fibroblasts’ Cholesterol Transport in Defective CHO Cells
$8,900Completed
1 7/1/1993 – 6/30/1997 Eugene Carstea, Ph.D.
(National Institutes of Health; Bethesda, Maryland, USA)
(5 years)
Identify the Gene Responsible for Niemann-Pick Type C Disease
$160,000Completed
More Details…