Research Fellowships
Research fellowships funded by NNPDF
Fellowship #22
Bilal Abdul Moiz
Peter G. Pentchev NPC Research Fellowship
University of Maryland, College Park, MD
PROJECT TITLE: Assessing the impact of membrane deficiency in Niemann-Pick Disease Type C on brain endotheilial-mediated glucose supply to the brain
PROJECT DATES: October 1, 2022 – October 1, 2023
PROJECT STATUS: Complete
Fellowship #21
Sara Naya Forcano
Edward H. Schuchman ASMD Research Fellowship
Fundacion Severo Ochoa, Madrid, Spain
PROJECT TITLE: Intranasal delivery of chitosan-encapsulated hydrocortisone to treat brain pathology in ASMD
PROJECT DATES: October 1, 2022 – October 1, 2023
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
PROJECT STATUS: Complete
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 – March 31, 2022
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: Complete
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
Peter G. Pentchev Research Fellowship
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:
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