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Fellowships Funded by the
NNPDF and CCNNPDF

(To see a listing of the Research Grants 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

14 a 10/2014 - 10/2016

Gizely Andrade
Sponsor: John Foxe, Ph.D.
(Albert Einstein College of Medicine; Bronx, NY)

Peter G. Pentchev Research Fellowship*

Developing Neurophysiological Outcome Measures for Treatment Assessment in Niemann-Pick type C (NPC) Disease.

Please find the published paper here.

In progress

October 2014
Project Summary
13 jiang xuntian 10/2014 - 10/2015

Xuntian Jiang, PhD
(Washington University; St. Louis, MO)

Peter G. Pentchev Research Fellowship*

Development of a diagnostic test and newborn screen for Niemann-Pick C disease

In progress

October 2014 Project Summary
12 KS 10/2014 - 10/2016

Kanagaraj Subramanian
Sponsor: William E. Balch
(The Scripps Research Institute; La Jolla, CA)

Peter G. Pentchev Research Fellowship*

Roadmap for Drug Development for Niemann-Pick Disease

In progress

October 2014 Project Summary
11

Deepti Gadi

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.

Complete

April 2014 Update

October 2013 Update

April 2013 Update

Project Summary Sept 2012

10

Stephanie Cologna
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.

In progress

April 2014 Update

October 2013 Update

April 2013 Update

Project Summary Sept 2012

9


Samaranch

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.

In progress

April 2014 Update

October 2013 Update

April 2013 Update

Project Summary Sept 2012

8


 

Andrew Munkacsi

9/2011 -
9/2012

(Year 1)

Extended:

2/2013 -

2/2014

(Year 2)

Andrew Munkacsi, Ph.D.
Sponsor: Stephen L. Sturley, Ph.D.
(Columbia University Medical Center;
New York, New York; USA ~ Year 1)

Victoria University of Wellington, New Zealand.

Peter G. Pentchev Research Fellowship*

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

$50,000 yr 1; $50,000 yr 2
Jointly sponsored by NNPDF and CCNNPDF

Complete

Final Report

Aug 2013 - March 2014
Update

October 2012 Update

April 2012 Update

Project Summary Sept 2011

 

7






Maetzel
9/2010 -
9/2013

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

 

 

Peter G. Pentchev Research Fellowship*

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

Completed

Published Article, May 2014

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)

 

 

 

 

 

 

Peter G. Pentchev Research Fellowship*

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



Fabrizio Vacca
4/2010 -
4/2012

Fabrizio Vacca, Ph.D.
Sponsor: Professor Jean Gruenberg
(University of Geneva, Switzerland)

 

 

 

Peter G. Pentchev Research Fellowship*

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
Ian Williams
9/2009 - 9/2011

Ian Williams, Ph.D.
Sponsor: Professor Fran Platt
(University of Oxford, UK)

 

Peter G. Pentchev Research Fellowship*

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)

Peter G. Pentchev Research Fellowship*

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)

Peter G. Pentchev Research Fellowship*

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)

Peter G. Pentchev Research Fellowship*

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

$80,500

Completed

More Details...

Mouse Model Grant to Jackson Laboratory

The NNPDF is pleased be able to provide $2500 to help defray the costs of processing the mouse model related to Niemann-Pick Disease (help by tradece potter).  The mouse model was created by Dr. Richard Pagano of the Mayo Clinic College of Medicine and is being processed by the Jackson Laboratory Repository.  

The nonprofit Jackson Laboratory Repository in Bar Harbor, Maine, serves as a centralized NIH-funded facility for mouse models used by the international biomedical research community, in both academic and nonprofit institutions. 

[Jan 20, 2010 mem]


Edward H. Schuchman, Ph.D. (Mount Sinai School of Medicine). Construction & Characterization of New Mouse Models for Types A & B Niemann-Pick.   Click here to read a September 2008 progress summary.    Click here to read the May 2009 progress summary.

Frances M. Platt, Ph.D. (Dept. of Pharmacology, University of Oxford).  Targeting sphingosine storage as a new therapeutic approach for Niemann-Pick Type C.  Click here to read the October 2008 progress summary.   Click here to read the March 2009 progress summary.      Click here to read the October 2009 progress summary.

Seymour Packman, MD, Director, UCSF-Stanford Lysosomal Disease Center
Underwritten by a grant from Genzyme Corporation

(To see a listing of Fellowships funded by the NNPDF, click here.)

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

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

[June 22, 2009 mem]

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