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Fellowships Jointly 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

17 01/2016 – 12/2018 Ofer Moldavski, PhD
(The University of California, Berkley, CA)Peter G. Pentchev Research Fellowship*
New Perspective on Niemann-Pick type C Disease as an mTORC1 Dependent Metabolic Disease

In progress

Jan 2016 Project Summary
16 jiang_xuntian 01/2016 – 12/2016 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

Jan 2016 Project Summary
15

AndrewMunkacsi

 

Extended:
05/2015 -11/2015
(Year 3)

 

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 Bridge Funding

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

$20,000 yr 3 extension

Jointly sponsored by NNPDF and CCNNPDF

In progress

May 2015 Project Summary

14 10577034_10152636003303856_4513193840679195561_n 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

3. October 2015 Project Summary

2. April 2015 Project Summary

1. 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

Complete

3. October 2015 Final Report

2. April 2015 Project Summary

1. 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

3. October 2015 Project Summary

2. April 2015 Project Summary

1. October 2014 Project Summary

11 DeeptiGadi 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 FINAL Update
October 2013 Update April 2013 Update

Project Summary Sept 2012

10
StephanieCologna_000
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.

Completed

Published Article, Sept 3rd, 2015

October 2014 FINAL Report

April 2014 Update

October 2013 Update

April 2013 Update

Project Summary Sept 2012

9 Luis 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.

Completed

October 2014 FINAL Report

April 2014 Update

October 2013 Update

April 2013 Update

Project Summary Sept 2012

8 AndrewMunkacsi

9/2011 –
9/2012

(Year 1)

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 Bridge Funding

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

Completed ~ see extension

April 2014 Final Report

Aug 2013 – March 2014
Update

October 2012 Update

April 2012 Update

Project Summary Sept 2011

7 maetzel_002 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 FabrizioVacca_000 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 UpdateApril 2011
Update
Project Summary Sept 2010
4 ianw 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…