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The Children's Fund for GSD Research is a not-for-profit (501c3) foundation, established to benefit children born with Glycogen Storage Disease (GSD1).
203.272.CURE
203.272.7744
Research Supported by Our Foundation
 

Institution

Researcher
Topic
Amount Granted
Date
NIH
Dr. Janice Chou
 Development of Gene Therapy Vectors for GSD 1a with improved efficacy
$147,220
2016
 

NIH

Dr. Janice Chou

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

$89,028

 

2015
 
GlyGenix Theurapedics, Inc.
Dr. William Fodor
Chemistry, Manufacturing and Controls: Ensuring AAV2/8-G6Phase is produced to meet FDA Standardss Suitable for Toxicology and Clinical Studies
$104,000
2014
 

University of Florida 

Dr. Nataly Clement

 

$38,368

 

2014
 
NIH
Dr. Janice Chou
Evaluation of the efficacy of of AAV8-GPW in GSD-1a mice with pre-existing hepotacellular adenoma
$106,306
2014

 

University of Florida

Dr. David Weinstein, Dr. Thomas Conlon

Gene Therapy for Glycogen Storage Disease, Type 1a

$156,083

 

2013
 
NIH
Dr. Janice Chou
Evaluation of the efficacy of a recombinant AAV vector expressing the condon-optimized human G6Phase
$98,996
2013
NIH

Dr. Janice Chou

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

$95,846

 

2012
 
University of Florida
Dr. Thomas Conlon, Dr. David Weinstein, Dr. Barry Byrne
Gene Therapy for GSD1
$117,508
2012

NIH

Dr. Janice Chou

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

$44,264.50

 

2012
Duke University
Dr. Dwight Koeberl
The Evaluation of the efficacy of two AAV vectors in murine GSD1a  
$55,016.50
2012

NIH

Dr. Janice Chou

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

$88,529

 

2011
Duke University
Dr. Dwight Koeberl
The Evaluation of the efficacy of two AAV vectors in murine GSD1a
$110,033
2011
NIH
Dr. Janice Chou
Long-term consequences of AAV-mediated gene therapy for GSD1a
$93,936
2011
Duke University
Dr. Dwight Koeberl
Pre-Clinical Development of AAV vectors for Gene Therapy in GSD1a
$167,087
2010
University of Florida

Dr. Thomas Conlon Dr. David Weinstein, Dr. Barry Byrne

Gene Therapy for GSD1a

$570,553

(over 2 years)

2010
University of Florida/Duke University
Dr. David Weinstein/Dr. Priya Kishnani
A multicenter, double blind crossover study of the dosing and efficiency of modified resistant cornstarch in GSD1a patients
$54,541
2009
University of Florida
Dr. Thomas Conlon/Dr. Catherine Mah
Gene Therapy for GSD1a
$100,000
2009
Duke Universtiy
Dr. Dwight Koeberl/Dr. Carlos Pinto
Pre-clinical development of a highly effecacious AAV vector in GSD1a dogs
$23,049
2009
NIH
Dr. Janice Chou

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

$74,030

November 2008
Stanford University
Dr. Howard Sussman

Identification of Pharmaceutical Targets for Hepatic Adenoma in GSD1a

$97,748

November 2008
Duke University
Dr. Dwight Koeberl

Development of AAV Vectors for GSD1a

$67,159

November

2008

Baylor College of Medicine
Dr. Lawrence Chan
Curative Treatment for GSD1a in a Dog Model

$183,222

(over 2 years)

September 2007
NIH
Dr. Janice Chou
Renal Disease in GSD 1a
$55,365
September 2007
University of Florida
Dr. Cathryn Mah
Gene Therapy for GSD1a

$499,538

(over 2 years)

September 2007
NC State College of Veterinary Medicine
Dr. Carlos Pinto
Pre-Clinical Development of a Highly Efficacious AAV Vector in GSD1a Dogs

$251,211

(over 2 years)

September 2007
Stanford University
Dr. Howard Sussman
Identification of Pharmaceutical Targets for Hepatic Adenoma in GSD1a
$95,091
September 2007

Duke University and University of Florida

Drs. Priya Kishnani and David Weinstein

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

$249,382

(over 15 months)

September 2007

University of Florida

College of Medicine

Dr. Bryon Peterson, PhD
Gene/Stem Cell Therapy for the correction of GSD1 Disease
$124,797
August, 2006
North Carolina State University, College of Veterinary Medicine
Dr. Carlos Pinto, DVM, PhD
Long-term correction of canine GSD1a using AAV2/8 vectors and toxicity of helper-dependent and AAV viral vectors
$86,049
August, 2006
Duke University Medical Center
Dr. Dwight Koeberl, MD, PhD
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)

August, 2006
Stanford University Medical Center
Dr. Howard Sussman, MD
Identification of pharmaceutical targets for hepatic adenoma in GSD1a

$130,724

(over 2 years)

August, 2006
National Institutes of Health, Bethesda, Maryland

Dr. Janice Chou and

Mohammad

Allamarvdasht

Recombinant adeno-associated virus-mediated gene therapy for glycogen storage disease type 1 directed by the human glucose-6-phosphatase promoter
$158,300
November, 2005
University of Florida, Dept. of Pediatrics
Dr. David Weinstein
An open-label, crossover study of alternative dietary therapies for GSD, Type 1
$153,442
November, 2005
Duke Children's Hospital and Health Center
Drs. Priya Kishnani and Deeksha Bali
Clinical and Molecular Evaluations in Glycogen Storage Disease Type 1a
$169,248
August, 2005
North Carolina State Univ., College of Veterinary Medicine
Dr. Talmage Brown
Long-term Correction of Canine GSD1a using AAV2/8 Vectors
$111,809
August, 2005
University of Florida, College of Medicine
Bryon Petersen, Ph.D
Gene/Stem Cell Therapy for the Correction of GSD1 Patients
$125,175
August, 2005
University of Florida, College of Medicine
Drs. Cathryn Mah and Barry Byrne
Gene Therapy for Glycogen Storage Disease Type 1a
$155,285.40
April, 2005

Goldyne Savad Institute of Gene Therapy, Hadassah University Hospital
Jerusalem, Israel

NIH
Bethesda, Maryland

Soroka Medical Center
Beersheva,
Israel

Drs. Reba Condiotti and Ethan Galun in collaboration with Drs. Janice Chou and Shimon Moses
Use of a non-primate lentiviral vector for stable and prolonged G6Pase espression for therapy of GSD1a
$145,000
August, 2004

Duke University Medical Center
North Carolina

Drs. Dwight Koeberl and Haoyue Zhang, Ayn Schneider and Andrew Bird
Regulated G6Pase expression with AAV vectors to correct GSD1a
$183,310

August, 2004

Beatrix Children's Hosptial, University Hospital
Groningen,

The Netherlands

Dr. G.P.A. Smit and Danielle Martens
ISGSD1 project and patient database
$32,400

August, 2004

Children's Hosptial Boston

Harvard Medical School

Boston

Drs. David Weintstein, Emanuela Gussoni, Mark Flemming and Morey Haymond
Quantification of Endogenous Glucose Production as a Marker of Glucose-6-Phosphatase Activity after Bone Marrow Transplantation
$76,558
August, 2004

Children's Hospital Boston

Drs. David Weinstein, David Breault, and William Tse
Use of Bone Marrow Derived Stem Cells to Correct Glucose-6-Phosphatase Activity in Type 1a Glycogen Storage Disease
$50,000
June 14, 2003

Duke University Medical Center
North Carolina

Drs. Dwight Koeberl and Talmage Brown, Jr.
The developement of gene therapy with AAV vectors in GSD1a
$50,000
June 20, 2003

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