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Fellowships Funded by the
National Niemann-Pick Disease Foundation

(To see a listing of the Research Grants funded by the CCNNPDF/NNPDF, click here .)

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

The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) is a sister chapter to the National Niemann-Pick Disease Foundation (NNPDF) (U.S.) and receives administrative support from the NNPDF. Money raised through the CCNNPDF is invested in Niemann-Pick Disease research through the NNPDF's research program with guidance from the NNPDF's Scientific Advisory Board (SAB), Board of Directors, and Research Committee. This partnership helps eliminate redundancy and maximizes the impact of research dollars. (Tammy Vaughan, chair of the CCNNPDF, serves as secretary of the NNPDF, and is on the NNPDF's Research Committee.)


The research funded by the NNPDF is made possible in great part due to the efforts of our NPD families in both the U.S. and Canada, and their extended support network via local community-sponsored events. The NNPDF is truly grateful for this support.

Item #


Date


Researcher


Project

Additional
Information

11

10/2012 - 10/2014

Deepti Gadi, Ph.D.
Sponsor: Frederick Maxfield, Ph.D.
(Weill College of Cornell University; New York, New York)


Peter G. Pentchev Research Fellowship*

Understanding the cellular mechanism of HDAC inhibitors for the treatment of NPC disease

Project Summary Sept 2012

April 2013 Update

10
10/2012 - 10/2014

Stephanie M. Cologna, Ph.D.
Sponsor: Forbes D. Porter, Ph. D.
(National Institute of Health; Bethseda, Maryland)


Peter G. Pentchev Research Fellowship*

Differential Protein Expression Profiling in Cerebrospinal Fluid from Niemann-Pick Disease, type C1 Patients

Project Summary Sept 2012

April 2013 Update

9

10/2012 - 10/2014

Lluis Samaranch Gusi, Ph.D.
Sponsor: Krystof S. Bankiewicz, M.D., Ph. D.
(University of California; San Francisco, California; USA)


Edward H. Schuchman Fellowship*

AAV9-mediated human acid sphingomyelinase expression in nonhuman primate brain: Preclinical development of gene therapy for Niemann-Pick disease type A

Project Summary Sept 2012

April 2013 Update

October 2013 Update

8

9/2011 -
9/2012

Andrew Munkacsi, Ph.D.
Sponsor: Stephen L. Sturley, Ph.D.
(Columbia University Medical Center;
New York, New York; USA)

Defining the molecular basis of HDAC inhibitors to treat murine, feline, and human models of NPC

$50,000
Jointly sponsored by NNPDF and CCNNPDF

In progress  - Possible extension

October 2012 Update

April 2012 Update

7





9/2010 -
9/2013
Dorothea Maetzel, Ph.D.
Sponsor: Rudolf Jaenisch, M.D.
(Whitehead Institute for Biomedical Research; Cambridge, Massachusetts; USA)

In Vitro modelling of Niemann-Pick type C Disease Using Patient-Specific Induced Pluripotent Stem Cells

$100,000
Jointly sponsored by NNPDF and CCNNPDF

Received third year extension of additional $50,000

In progress

Final Lay Summary Report

March - August 2012 Update

April 2012 Update

Oct 2011 Update

April 2011 Update

Project Summary Sept 2010

6
9/2010 -
9/2013
Nicholas Cianciola, Ph.D.
Sponsor: Cathleen Carlin, Ph.D.
(School of Medicine, Case Western Reserve University; Cleveland, Ohio; USA)

Activation of an alternative cholesterol homeostatic mechanism in NPC

$100,000
Jointly sponsored by NNPDF and CCNNPDF

Received third year extension of additional $50,000

Completed

Final Lay Summary

April 2013 Update

Oct 2012 Update

April 2012 Update

Oct 2011 Update

April 2011 Update

Project Summary Sept 2010

5



4/2010 -
4/2012
Fabrizio Vacca, Ph.D.
Sponsor: Professor Jean Gruenberg
(University of Geneva, Switzerland)
Analysis of cholesterol export from purified endosomes in NPC cellular models

$100,000
Jointly sponsored by NNPDF and CCNPDF

Completed

Final Lay Summary Report
June 2012

Oct 2011 Update

April 2011
Update


Project Summary Sept 2010

4
9/2009 - 9/2011 Ian Williams, Ph.D.
Sponsor: Professor Fran Platt
(University of Oxford, UK)

Neurobiology of Purkinje Cells in NPC1

$100,000
Jointly sponsored by NNPDF and CCNNPDF

Completed

Oct 2011 Update

April 2011 Update

Project Summary Sept 2010

3

9/15/2004 - 9/14/2006

Nobutaka Ohgami, Ph.D.
Sponsor: T. Y. Chang, Ph.D.
(Dartmouth Medical School; Hanover, New Hampshire; USA)
(2)

Demonstration of NPC1 as a lipid binding protein in Vitro

$80,500

contract modified Jan 2006 to end 31/12/2007

Completed

More Details...

2

3/15/2003 - 3/14/2005

Heng-Ling Liou, Ph.D.
Sponsor: Peter Lobel, Ph.D.
(University of Medicine & Dentistry of New Jersey -Robert Wood Johnson Medical School; Piscataway, New Jersey, USA)
(2 years)

The Role of NPC2 (Niemann-Pick Disease Type C2) Protein in Lysosomal Cholesterol Trafficking

$80,500

Completed

More Details...

1

3/15/2003 - 3/14/2005

Amit Kumar Choudhury, Ph.D.
Sponsor: Richard E. Pagano, Ph.D.
(Mayo Clinic and Foundation; Rochester, Minnesota, USA)
(2 years)

Reduction of Lipid Accumulation in Niemann-Pick Fibroblasts by Modulation of Membrane Trafficking

$80,500

Completed

More Details...

“The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the Canadian Chapter of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the CCNNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.”