RESEARCH SUPPORTED BY OUR FOUNDATION

Our foundation has granted over $8 million, leading to groundbreaking therapies. 

We have supported almost 60 studies to help scientists investigate probable approaches to improve treatment and develop a cure.

ALL RESEARCH STUDIES SUPPORTED BY OUR FOUNDATION

Below is a comphrensive list of research we have funding since we began in 2002.  If you are interested in applying for a grant, please download the application guidelines.

Studies Funded in 2021

  • .
  • Researcher
  • Institution
  • 2021 Granted Research Projects
  • Dr. Janice Chou
  • NIH

  • Evaluate the efficiency of CRISPR/Cas9-based gene editing system to correct G6PC mutation in an animal model of GSD-1a.

    $186,720.00

    $186,720

    $186,720.00 

  • Dr. Youngmok Lee
  • University of Connecticut

  • Development of kidney direct gene therapy and research to improve the efficacy of AAV mediated gene therapy in GSD-1a mice.

    $195,845.00

Studies Funded in 2019

  • .
  • Researcher
  • Institution
  • 2019 Granted Projects
  • Dr. Junho Cho
  • University of Connecticut

  • Evaluation of the strategy for autophagy normalization to prevent HCA/HCC in glycogen storage disease type 1a.

    $199,056.00

  • Dr. Youngmok Lee
  • University of Connecticut

  • Exosome marker development for hepatocellular adenoma in human GSD1a & Development of kidney direct gene therapy in GSD-1a mice.

    $357,667.00

  • Dr. Janice Chou
  • NIH

  • Evaluate the efficiency of CRISPR/Cas9-based gene editing system to correct a G6PC mutation in an animal model of GSD 1a.

    $226,720.00

  • Heather Saavedra
  • University of Texas

  • Support of gene therapy trials

    $7,200.00

  • Dr. David Weinstein
  • University of Connecticut

  • Support of gene therapy trials

    $15,000.00

  • Dr. Ayesha Ahmad
  • University of Michigan

  • Support of gene therapy trials

    $7,200.00

Studies Funded in 2018

  • .
  • Researcher
  • Institution
  • 2018 Granted Projects
  • Dr. Youngmok Lee
  • University of Connecticut

  • Exosome marker development for hepatocellular adenoma in human GSD1a

    $37,039.00

  • Dr. David Weinstein
  • University of Connecticut

  • Evaluation of the efficacy of gene therapy to arrest or abrogate the preexisting hepatocellular adenoma/carcenoma in glycogen storage disease type 1a

    $153,011.00

  • Dr. Janice Chou
  • NIH

  • Evaluate the efficacy of CRISPR/Cas9-based gene editing system to correct a G6PC mutation in an animal model of GSD 1a

    $160,040.00

  • Dr. Youngmok Lee
  • University of Connecticut

  • Research development to improve the efficacy of AAV mediated gene therap in GSD1a mouse model

    $370,326 (over 2 years)

  • Dr. Mitchell Drumm
  • Case Western Reserve University

  • Various GSD 1a studies

    $203,540.00

  • Dr. David Weinstein
  • University of Connecticut

  • Evaluation of the efficacy of gene therapy to arrest or abrogate the preexisting hepatocellular adenoma/carcinoma in glycogen storage disease type 1a. Colony

    $153,011.00

Studies Funded in 2017

  • .
  • Researcher
  • Institution
  • 2017 Granted Projects
  • Dr. Thomas Conlon
  • University of Florida

  • Colony maintenance of glycogen storage disease type 1a canines treated with AAV gene therapy

    $121,395.00

  • Dr. Janice Chou
  • NIH

  • Evaluate the efficiency of CRISPR/Cas9-based gene editing system to correct a G6PC mutation in an animal model of GSD 1a

    $152,850.00

  • Dr. Youngmok Lee
  • Univesity of Connecticut

  • Development of kidney direct gene therapy in GSD 1a mice

    $158,329.00

Studies Funded in 2016

  • .
  • Researcher
  • Institution
  • 2016 Granted Projects
  • Dr. Janice Chou
  • NIH

  • Development of Gene Therapy Vectors for GSD 1a with improved efficacy

    $147,220.00

Studies Funded in 2015

  • .
  • Researcher
  • Institution
  • 2015 Granted Projects
  • Dr. Janice Chou
  • NIH

  • Evaluation of the efficacy of AAV8-GPE in GSD-la mice with pre-existing hepatocellular adenoma

    $89,020.00

Studies Funded in 2014

  • .
  • Researcher
  • Institution
  • 2014 Granted Projects
  • Dr. William Fodor
  • GlyGenix Theurapedics, Inc.
  • Chemistry, Manufacturing and Controls: Ensuring AAV2/8-G6Phase is produced to meet FDA Standardss Suitable for Toxicology and Clinical Studies

    $104,000.00

  • Dr. Nataly Clement
  • University of Florida

  • $38,368.00

  • Dr. Janice Chou
  • NIH

  • Evaluation of the efficacy of of AAV8-GPW in GSD-1a mice with pre-existing hepotacellular adenoma

    $106,306.00

Studies Funded in 2013

  • .
  • Researcher
  • Institution
  • 2013 Granted Projects
  • Dr. David Weinstein, Dr. Thomas Conlon
  • University of Florida

  • Gene Therapy for Glycogen Storage Disease, Type 1a

    $156,083.00

  • Dr. Janice Chou
  • NIH

  • Evaluation of the efficacy of a recombinant AAV vector expressing the condon-optimized human G6Phase

    $98,996.00

Studies Funded in 2012

  • .
  • Researcher
  • Institution
  • 2012 Granted Projects
  • Dr. Janice Chou
  • NIH

  • Evaluation of the efficacy of AAV8-GPE in GSD1a mice with pre-existing hepatocelluar adenoma

    $95,846.00

  • Dr. Thomas Conlon, Dr. David Weinstein, Dr. Barry Byrne
  • University of Florida

  • Gene Therapy for GSD1

    $117,508.00

  • Dr. Janice Chou
  • NIH

  • The Evaluation of the efficacy of two AAV vectors in murine GSD1a

    $44,264.50

  • Dr. Dwight Koeberl
  • Duke University

  • The Evaluation of the efficacy of two AAV vectors in murine GSD1a

    $55,016.50

Studies Funded in 2011

  • .
  • Researcher
  • Institution
  • 2011 Granted Projects
  • Dr. Janice Chou
  • NIH

  • The Evaluation of the efficacy of two AAV vectors in murine GSD1a

    $88,529.00

  • Dr. Dwight Koeberl
  • Duke University

  • The Evaluation of the efficacy of two AAV vectors in murine GSD1a

    $110,033.00

  • Dr. Janice Chou
  • NIH

  • Long-term consequences of AAV-mediated gene therapy for GSD1a

    $93,936.00

Studies Funded in 2010

  • .
  • Researcher
  • Institution
  • 2010 Granted Projects
  • Dr. Dwight Koeberl
  • Duke University

  • Pre-Clinical Development of AAV vectors for Gene Therapy in GSD1a

    $167,087.00

  • Dr. Thomas Conlon Dr. David Weinstein, Dr. Barry Byrne
  • University of Florida

  • Gene Therapy for GSD1a

    $570,553(over 2 years)

Studies Funded in 2009

  • .
  • Researcher
  • Institution
  • 2009 Granted Projects
  • Dr. David Weinstein/Dr. Priya Kishnani
  • University of Florida/Duke University

  • A multicenter, double blind crossover study of the dosing and efficiency of modified resistant cornstarch in GSD1a patients

    $54,541.00

  • Dr. Thomas Conlon/Dr. Catherine Mah
  • University of Florida

  • Gene Therapy for GSD1a

    $100,000.00

  • Dr. Dwight Koeberl/Dr. Carlos Pinto
  • Duke Universtiy

  • Pre-clinical development of a highly effecacious AAV vector in GSD1a dogs

    $23,049.00

Studies Funded in 2008

  • .
  • Researcher
  • Institution
  • 2008 Granted Projects
  • Dr. Janice Chou
  • NIH

  • Long-term consequences of AAV-mediated gene therapy for GSD-Ia.

    $74,030.00

  • Dr. Howard Sussman
  • Stanford University

  • Identification of Pharmaceutical Targets for Hepatic Adenoma in GSD1a

    $97,748.00

  • Dr. Dwight Koeberl
  • Duke Universtiy

  • Development of AAV Vectors for GSD1a

    $67,159.00

Studies Funded in 2007

  • .
  • Researcher
  • Institution
  • 2007 Granted Projects
  • Dr. Lawrence Chan
  • Baylor College of Medicine

  • Curative Treatment for GSD1a in a Dog Model

    $183,222 (over 2 years)

  • Dr. Janice Chou
  • NIH

  • Renal Disease in GSD 1a

    $55,365.00

  • Dr. Cathryn Mah
  • University of Florida

  • Gene Therapy for GSD1a

    $499,538 (over 2 years)

  • Dr. Carlos Pinto
  • NC State College of Veterinary Medicine

  • Pre-Clinical Development of a Highly Efficacious AAV Vector in GSD1a Dogs

    $251,211 (over 2 years)

  • Dr. Howard Sussman
  • Stanford University

  • Identification of Pharmaceutical Targets for Hepatic Adenoma in GSD1a

    $95,091.00

  • Drs. Priya Kishnani and David Weinstein
  • Duke University and University of Florida

  • A multicenter, double-blind crossover study of the dosing and efficiency of modified resistant cornstarch in patients with GSD1a.

    $249,382 (over 15 months)

Studies Funded in 2006

  • .
  • Researcher
  • Institution
  • 2006 Granted Projects
  • Dr. Bryon Peterson, PhD
  • University of Florida College of Medicine

  • Gene/Stem Cell Therapy for the correction of GSD1 Disease

    $124,797.00

  • Dr. Carlos Pinto, DVM, PhD
  • North Carolina State University, College of Veterinary Medicine

  • Long-term correction of canine GSD1a using AAV2/8 vectors and toxicity of helper-dependent and AAV viral vectors

    $86,049.00

  • Dr. Dwight Koeberl, MD, PhD
  • Duke University Medical Center

  • Preclinical Development of AAV vectors for gene therapy in GSD1a and the evaluation of the long-term safety and efficacy of AAV vectors in the available animal models.

    $157,658 (over 2 years)

  • Dr. Howard Sussman, MD
  • Stanford University Medical Center

  • Identification of pharmaceutical targets for hepatic adenoma in GSD1a

    $130,724 (over 2 years)

Studies Funded in 2005

  • .
  • Researcher
  • Institution
  • 2005 Granted Projects
  • Dr. Janice Chou and Mohammad Allamarvdasht
  • National Institutes of Health, Bethesda, Maryland

  • Recombinant adeno-associated virus-mediated gene therapy for glycogen storage disease type 1 directed by the human glucose-6-phosphatase promoter

    $158,300.00

  • Dr. David Weinstein
  • University of Florida, Dept. of Pediatrics

  • An open-label, crossover study of alternative dietary therapies for GSD, Type 1

    $153,442.00

  • Drs. Priya Kishnani and Deeksha Bali
  • Duke University Medical Center

  • Clinical and Molecular Evaluations in Glycogen Storage Disease Type 1a

    $169,248.00

  • Dr. Talmage Brown
  • North Carolina State Univ., College of Veterinary Medicine

  • Long-term Correction of Canine GSD1a using AAV2/8 Vectors

    $111,809.00

  • Bryon Petersen, Ph.D
  • University of Florida, College of Medicine

  • Gene/Stem Cell Therapy for the Correction of GSD1 Patients

    $125,175.00

  • Drs. Cathryn Mah and Barry Byrne
  • University of Florida, College of Medicine

  • Gene Therapy for Glycogen Storage Disease Type 1a

    $155,285.40

Studies Funded in 2004

  • .
  • Researcher
  • Institution
  • 2004 Granted Projects
  • Drs. Reba Condiotti and Ethan Galun in collaboration with Drs. Janice Chou and Shimon Moses
  • Goldyne Savad Institute of Gene Therapy, Hadassah University Hospital Jerusalem, Israel NIH Bethesda, Maryland Soroka Medical Center Beersheva, Israel

  • Use of a non-primate lentiviral vector for stable and prolonged G6Pase espression for therapy of GSD1a

    $145,000.00

  • Drs. Dwight Koeberl and Haoyue Zhang, Ayn Schneider and Andrew Bird
  • Duke University Medical Center North Carolina

  • Regulated G6Pase expression with AAV vectors to correct GSD1a

    $183,310.00

  • Dr. G.P.A. Smit and Danielle Martens
  • Beatrix Children's Hosptial, University Hospital Groningen, The Netherlands

  • ISGSD1 project and patient database

    $32,400.00

  • Drs. David Weintstein, Emanuela Gussoni, Mark Flemming and Morey Haymond
  • Children's Hosptial Boston Harvard Medical School Boston

  • Quantification of Endogenous Glucose Production as a Marker of Glucose-6-Phosphatase Activity after Bone Marrow Transplantation

    $76,558.00

Studies Funded in 2003

  • .
  • Researcher
  • Institution
  • 2003 Granted Projects
  • Drs. David Weinstein, David Breault, and William Tse
  • Children's Hospital Boston

  • Use of Bone Marrow Derived Stem Cells to Correct Glucose-6-Phosphatase Activity in Type 1a Glycogen Storage Disease

    $50,000.00

  • Drs. Dwight Koeberl and Talmage Brown, Jr.
  • Duke University Medical Center North Carolina

  • The developement of gene therapy with AAV vectors in GSD1a

    $50,000.00

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