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Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

2008-2002 Grants

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Resulting Publications

2008
2003
2002

Research Initiative Grants
2008
2007
2004-2005
2003
2002

2008 Research Initiative Grants

Angela Gritti, PhD and Alessandra Biffi, PhD
San Raffaele Telethon Institute for Gene Therapy
“Evaluation of Combined Approaches Using Hematopoietic and Neural Stem Cells for the Treatment of Globoid Cell Leukodystrophy

Stephanos Kyrkanides, PhD
Stony Brook University
“Retrograde transfer of therapeutic vectors enabled by the trigeminal sensory system”

2008 Research Grants - Resulting Publications

Gentner, S, et al., Therapy of Globoid Cell Leukodystrophy Sci Transl Med 2, 58ra84 (2010)

Kyrkanides, S, et al., The trigeminal retrograde transfer pathway in the treatment of Neurodegeneration, Journal of Neuroimmunology, 209 (2009) 139–142 pdf Journal of Neuroimmunology Pathway Treatment Neuro Kyrkanides 2009 (1.08 MB) (.pdf)

2007 Research Initiative Grants

Susan L. Cotman, PhD
Massachusetts General Hospital
“Small molecule screening to identify modifiers of lysosomal trafficking, a putative therapy for Batten”

Doug Martin, PhD
Auburn University
“Pre-clinical gene therapy for GM2 in a feline model”

Miguel Esteves, PhD
Massachusetts General Hospital
“AAV-mediated gene therapy for Tay-Sachs: Vector selection for pre-clinical development”

Aryan Namoodiri, PhD
Uniformed Services University of the Heath Sciences
* “Preclinical Research toward Acetate Supplementation Therapy for Canavan Disease”

This grant was made possible by through the generous support of NTSAD Members with children affected by Canavan and the Research Initiative Fund.

*This project was recently awarded significant funding in a multi-year NIH grant – congratulations!

Later in the year, multi-year grants were awarded to the Tay-Sachs Gene Therapy Consortium:

Florian Eichler, MD
Massachusetts General Hospital
“The Natural History of Tay-Sachs Disease”

Miguel Sena-Esteves, PhD
Massachusetts General Hospital
“AAV-mediated Gene Therapy for Tay-Sachs Disease: Vector Selection for Preclinical Development “

Timothy Cox, MD and M. Begoña Cachón-González, PhD
University of Cambridge
“Pre-Clinical/Clinical Research Program: Tay-Sachs and Related Diseases “

Douglas Martin, PhD
Auburn University
“Pre-Clinical Studies of AAV Gene Therapy in Feline GM2 Gangliosidosis”

Thomas Seyfried, PhD
Boston College
“Neurochemical and Immunological Evaluation of AAV Gene Therapy Strategies”

2004 - 2005 Research Initiative Grants

James A. Shayman, M.D.
University of Michigan
“High throughput screening for inhibitors of ganglioside GM2 synthase”

2003 Research Initiative Grants

Jean-Pyo Lee, Ph.D./Evan Y. Snyder lab
Beth Israel Deaconess Medical Center/Burnham Institute
“Therapeutic Potential of Neural Stem Cells in the Gangliosidoses (Tay-Sachs & Sandhoff Diseases)”

Cynthia Tifft, M.D., Ph.D., FACMG
Children’s Research Institute of Children’s National Medical Center
“Comprehensive Biochemical Analysis of Cerebrospinal Fluid in Patients with GM2 Storage Disorders:
Molecular Pathogenesis of Disease Progression”

2003 Research Grants - Resulting Publications

Lee J-P, Taylor RM, Platt F, Snyder EY, Neural stem cell therapies & applications to lysosomal storage disorder in J.A. Barranger, M.A. Cabrera-Salazar (Eds), Lysosomal Storage Diseases, Plenum Press (in press).

Lee et all. Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease. Nature Medicine, March 2007.   pdf Nature Medicine 2007 Stem Cells Multiple Mechanisms Lee Seyfried Platt Snyder (581 KB) (.pdf)

2002 Research Initiative Grants

Bruce A. Bunnell, Ph.D.
Tulane University Health Sciences Center
“In utero Gene Therapy of Sandhoff Disease in a Murine Model”

Stephanos Kyrkanides, D.D.S., M.S., Ph.D.
University of Rochester School of Medicine & Dentistry
*"Perinatal Gene Therapy for ?-hexosaminidase disorders (Tay-Sachs and Sandhoff diseases)”
* This project was later awarded significant funding in a multi-year NIH grant – congratulations!

Paola Leone, Ph.D.
University of Medicine and Dentistry of New Jersey
"Neuroprotective Effect of Minocycline in Sandhoff Disease"

Professor Thomas N. Seyfried, MS, Ph.D.
Boston College
“Therapeutic evaluation of NB-DGJ for ganglioside storage diseases”

2002 Research Grants - Resulting Publications

Kasperzyk et al. N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis. J. Neurochem. 89: 645-653, (2004).   pdf Journal of Neurochemistry GM1 Platt Seyfried Kasperzyk (140 KB) (.pdf)

Hauser et al. Inheritance of lysosomal acid beta-galactosidase activity and gangliosides in crosses of DBA/2J and knockout mice. Biochem. Genetics, 42: 241-257, (2004).   www.ncbi.nlm.nih.gov

Baek et al. Poster Session DP2: Gangliosides: N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice. J. Neurochem. 90: (Suppl. 1) 89, (2004).   pdf Poster Session DP2 Gangliosides (63 KB) (.pdf)

Denny et al. Poster Session PSM06: Metabolism: Retinal glycosphingolipid abnormalities in Sandhoff and GM1 gangliosidosis mice. J. Neurochem. 94 (s1), 39-43. (2005).   pdf Poster Session PSM06 Metabolism (344 KB) (.pdf)

Denny et al. Caloric restriction extends longevity without altering brain lipid composition or cytoplasmic neuronal vacuoles in Sandhoff mice. J. Neurochem. 94 (s1), 39-43. (2005). pdf Poster Session PSM06 Metabolism (344 KB) (.pdf)

Baek et al. Neural stem cell transplantation reduces brain GM2 and GA2 content in a mouse model of Sandhoff disease. J. Neurochem. 94 (s1), 23. (2005). pdf Poster Session PSM06 Metabolism (344 KB) (.pdf)

Baek et al. N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice. J. Neurochem. 90: (Suppl. 1) 89, (2004).

Kyrkanides et al. B-hexosaminidase lentiviral vectors: transfer into the CNS via systemic administration Molecular Brain Research (2005) 133: 286-298.   pdf Molecular Brain Research Kyrkanides et al 2005 (846 KB) (.pdf)

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