CGRF’s Research Projects

The Children’s Gaucher Research Fund makes grants to accredited institutions doing relevant research on Gaucher Disease Type 2 and/or Type 3.

To date, CGRF has raised over 2 million dollars. 100% of every dollar we receive goes to medical research.

When we created this nonprofit to honor our son’s life and the lives of all children affected by Gaucher Disease, we made the commitment to put all donations toward research. There are no salaries. There are no administrative costs. All costs for postage, printing, web site development, etc. are either donated or paid for by the founders. We express our deep gratitude to all of our contributors — people like you, who empower the research.

CGRF makes grants based on recommendations by our Scientific Advisory Board and by unaffiliated researchers around the world. Grant applications are ranked according to the following criteria:

  • Historical excellence demonstrated by the researcher and the associated medical institution.
  • Relevance of the proposed research grant to Type 2 or Type 3 Gaucher Disease.
  • Demonstration of efficiency regarding the proposed use of funds.

Research grant applications will be accepted from any accredited research institution in the world.

CGRF Grant Recipients

CGRF has awarded grants for the following research:

2002

“Molecular Mechanisms of Neuronal Dysfunction in Type 2 and Type 3 Gaucher Disease”
Dr. Tony Futerman
Weizmann Institute of Science – Israel

2003 – 2004

“Molecular Mechanisms of Neuronal Dysfunction in Type 2 and Type 3 Gaucher Disease”
Dr. Tony Futerman
Weizmann Institute of Science – Israel

2006

“The Role of Defective Calcium homeostasis in the neuropathology of type 2 and type 3 Gaucher disease: Deliniation of up and down-stream biochemical pathways”
Dr. Tony Futerman
Weizmann Institute of Science — Israel

2007

“Development of a model for Type 2 Gaucher Disease”
Dr. Lorne A. Clarke
Children’s and Woman’s Health Center of British Columbia — Canada

2007 – 2010

“The Role of Defective Calcium homeostasis in the neuropathology of type 2 and type 3 Gaucher disease: Deliniation of up and down-stream biochemical pathways”
Dr. Tony Futerman
Weizmann Institute of Science — Israel

2008 – 2012

“The Generation of an Inducible Neuronopathic Gaucher Disease Mouse Model”
Dr. Avraham Yaron
Dr. Yael Pewzner-Jung
Dr. Tony Futerman Ph.D.
Weizmann Institute of Science — Israel

 

Results of Research

Below is a partial list of published papers resulting from the work of our grant recipients:

Buccoliero, R. and Futerman, A.H. (2003) The roles of ceramide and complex sphingolipids in neuronal cell function. Pharamacol. Res. (Italy), 47, 409-419

Lloyd-Evans, E., Pelled, D., Riebeling, C., Bodennec, J., de-Morgan, A., Waller, H., Schiffmann, R., and Futerman, A.H. (2003)
Glucosylceramide and glucosylsphingosine modulate calcium mobilization from brain microsomes via different mechanisms. J. Biol. Chem., 278, 23594-23599.

Bodennec, J., Trajkovic-Bodennec, S. and Futerman, A.H. (2003) Simultaneous quantification of lyso-neutral-glycosphingolipids and neutral glycosphingolipids by N-acetylation with [3H]acetic anhydride. J. Lipid. Res., 44, 1413-1419.

Ginzburg, L., Kacher, Y. and Futerman, A.H. (2004) The pathogenesis of glycosphingolipid storage disorders. Sem. Cell Develop. Biol., 15, 417-431.

Pelled, D., Trajkovic-Bodennec, S., Lloyd-Evans, E., Sidransky, E., Schiffmann, S. and Futerman, A.H. (2005) Enhanced calcium release in the acute neuronopathic form of Gaucher disease. Neurobiol. of Disease, 18, 83-88.

Farfel-Becker, T., Vitner, E., Dekel, H., Leshem, N., Berglin-Enquist, I., Karlsson, S. and Futerman. A.H. (2009) No evidence for activation of the unfolded protein response in neuronopathic models of Gaucher disease. Hum. Mol. Genet., 8,1482-1488.

Vitner, E., Dekel, H., Zigdon, H., Shachar, T., Farfel-Becker, T., Eilam, R., Karlsson, S. and Futerman, A.H. (2010) Altered expression and distribution of cathepsins in neuronopathic forms of Gaucher disease and in other sphingolipidoses. Hum. Mol. Genet., 19, 3583-3590.

Farfel-Becker, T. and Futerman, A.H. (2010) Cellular pathogenesis in sphingolipid storage disorders: the quest for new therapeutic approaches. Clinical Lipidology, 5, 255-265.

Vitner, E., Platt, F.M. and Futerman, A.H. (2010) Common and uncommon pathogenic cascades in lysosomal storage diseases. J. Biol. Chem., 285, 20423-20427.