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Research Fellowships

Research fellowships funded by NNPDF

Fellowship #20

Steven Erwood

Peter G. Pentchev NPC Research Fellowship

The Hospital for Sick Children, Toronto (Canada)

PROJECT TITLE:  Generation of humanized mouse models for Niemann Pick disease Type C

PROJECT DATES:  September 1, 2020 – August 31, 2021

Fellowship #19

Christian Kappe, PhD Student

Edward H. Schuchman ASMD Research Fellowship

Humboldt University of Berlin (Germany)

PROJECT TITLE:  In celluluo characterization of small molecule activators of acid sphingomyelinase

PROJECT DATES:  September 1, 2020 – August 31, 2021

Fellowship #18

Adam Kanack

Peter G. Pentchev Research Fellowship The Medical College of Wisconsin PROJECT TITLE: Prevention of NPC1 Ubiquitination as a Therapeutic Target for Niemann-Pick Type C Disease PROJECT DATES:  October 2016 – October 2019 PROJECT STATUS:  In Progress PROJECT LAY SUMMARIES:

Fellowship #17

Ofer Moldavski, PhD

Peter G. Pentchev Research Fellowship

The University of California, Berlkey, CA

PROJECT TITLE: New Perspective on Niemann-Pick type C Disease as an mTORC1 Dependent Metabolic Disease

PROJECT DATES:  January 2016 – December 2018

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:

Fellowship #16

Xuntian Jiang, PhD

Peter G. Pentchev Research Fellowship

Washington University, St. Louis, MO

PROJECT TITLE: Development of a Diagnostic Test and Newborn Screen for Niemann-Pick C Disease

PROJECT DATES:  January 2016 – January 2017

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:

Fellowship #15

Andrew Munkacsi, PhD

Peter G. Pentchev Research Bridge Funding

Columbia University Medical Center, New York, NY (Year 1)

Victoria University of Wellington, New Zealand

PROJECT TITLE: Defining the Molecular Basis of HDAC Inhibitors to treat Murine, Feline, and Human Models of NPC

PROJECT DATES:  Extended May 2015 – November 2015 (Year 3)

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


Harnessing Hope to Treat Rare Disease in Children Article
(Press Release from Victoria University)


Vorinostat Published Paper in The Journal of Biological Chemistry


SPONSOR: Stephen L. Sturley, PhD
$20,000 Year 3 Extension
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #14

Gizely Andrade

Peter G. Pentchev Research Fellowship

Albert Einstein College of Medicine, Bronx, NY

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

PROJECT DATES:  October 2014 – October 2016

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


Sponsor: John Foxe, Ph.D.

Fellowship #13

Xuntian Jiang, PhD

Peter G. Pentchev Research Fellowship

Washington University, St. Louis, MO

PROJECT TITLE: Development of a Diagnostic Test and Newborn Screen for Niemann-Pick C Disease

PROJECT DATES:  October 2014 – October 2015

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:

Fellowship #12

Kanagaraj Subramanian

Peter G. Pentchev Research Fellowship

The Scripps Research Institute, La Jolla, CA

PROJECT TITLE: Roadmap for Drug Development for Niemann-Pick disease

PROJECT DATES:  October 2014 – October 2016

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


Sponsor: William E. Balch

Fellowship #11

Deepti Gadi, PhD

Peter G. Pentchev Research Fellowship

Weill College of Cornell University, New York, NY

PROJECT TITLE: Understanding the Cellular Mechanism of HDAC Inhibitors for the Treatment of NPC Disease

PROJECT DATES:  October 2012 – October 2014

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


Sponsor: Frederick Maxfield, PhD

Fellowship #10

Stephanie M. Cologna, PhD

Peter G. Pentchev Research Fellowship

National Institute of Health, Bethseda, MD

PROJECT TITLE: Differential Protein Expression Profiling in Cerebrospinal Fluid from Niemann-Pick disease, type C1 Patients

PROJECT DATES:  October 2012 – October 2014

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


September 2015 | Published Article

Sponsor: Forbes D. Porter, PhD

Fellowship #9

Lluis Samarach Gusi, PhD

Edward H. Schuchman Fellowship

University of California, San Francisco, CA

PROJECT TITLE: AAV9-Mediated Human Acid Sphingomyelinase Expression in Nonhuman Primate Brain: Preclinical Development of Gene Therapy for Niemann-Pick disease Type A

PROJECT DATES:  October 2012 – October 2014

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


Krystof S. Bankiewicz, MD, PhD

Fellowship #8

Andrew Munkacsi, PhD

Peter G. Pentchev Research Bridge Funding

Columbia University Medical Center, New York, NY (Year 1)
Victoria University of Wellington, New Zealand

PROJECT TITLE: Defining the Molecular Basis of HDAC Inhibitors to treat Murine, Feline, and Human Models of NPC

PROJECT DATES:  September 2011 – September 2012 (Year 1)
February 2013 – February 2014

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: Stephen L. Sturley, PhD
$50,000 Year 1; $50,000 Year 2
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #7

Dorothea Maetzel, PhD

Whitehead Institute for Biomedical Research, Cambridge, MA

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

PROJECT DATES:  September 2010 – September 2013

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


May 2014 | Published Article

SPONSOR: Rudolf Jaenisch, MD
$100,000
Jointly Sponsored by NNPDF and CCNNPDF
Received third year extension of additional $50,000

Fellowship #6

Nicholas Cianciola, PhD

Peter G. Pentchev Research Fellowship

School of Medicine, Case Western Reserve University, Cleveland, OH

PROJECT TITLE: Activation of an Alternative Cholesterol Homeostatic Mechanism in NPC

PROJECT DATES:  September 2010 – September 2013

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: Cathleen Carlin, PhD
$100,000
Jointly Sponsored by NNPDF and CCNNPDF
Received third year extension of additional $50,000

Fellowship #5

Fabrizio Vacca, PhD

Peter G. Pentchev Research Fellowship

University of Geneva, Switzerland

PROJECT TITLE: Analysis of Cholesterol Export from Purified Endosomes in NPC Cellular Models

PROJECT DATES:  April 2010 – April 2012

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: Professor Jean Gruenberg
$100,000
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #4

Ian Williams, PhD

Peter G. Pentchev Research Fellowship

University of Oxford, UK

PROJECT TITLE: Neurobiology of Purkinje Cells in NPC1

PROJECT DATES:  September 2009 – September 2011

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: Professor Fran Platt
$100,000
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #3

Nobutaka Ohgami, PhD

Peter G. Pentchev Research Fellowship

Dartmouth Medical School,  Hanover, NH

PROJECT TITLE: Demonstration of NPC1 as a Lipid Binding Protein in Vitro

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

PROJECT DATES:  September 2004 – September 2006

CONTRACT MODIFIED: January 2006 to December 2007

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: T.Y. Chang, PhD
$80,500
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #2

Heng-Ling Liou, PhD

Peter G. Pentchev Research Fellowship

University of Medicine & Dentistry of New Jersey -Robert Wood Johnson Medical School, Piscataway, NJ

PROJECT TITLE: The Role of NPC2 (Niemann-Pick disease Type C2) Protein in Lysosomal Cholesterol Trafficking

PROJECT DATES:  March 2003 – March 2005

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: Peter Lobel, PhD
$80,500
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #1

Amit Kumar Choudhury, PhD

Peter G. Pentchev Research Fellowship

Mayo Clinic and Foundation; Rochester, MN

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

PROJECT DATES:  March 2003 – March 2005

PROJECT STATUS:  Complete

PROJECT LAY SUMMARIES:


SPONSOR: Richard E. Pagano, PhD
$80,500
Jointly Sponsored by NNPDF and CCNNPDF

Fellowship #18

Adam Kanack

Peter G. Pentchev Research Fellowship

The Medical College of Wisconsin

PROJECT TITLE: Prevention of NPC1 Ubiquitination as a Therapeutic Target for Niemann-Pick Type C Disease

PROJECT DATES: October 2016 – October 2019

PROJECT STATUS: In Progress

PROJECT LAY SUMMARIES:

Supporting one another. Supporting our community.