Medical
Research and Treatments
Breakthroughs in gene replacement
therapy have resulted from our efforts, that are not only improving the quality
of life of children battling Canavan disease, but also giving hope for more
effective treatments of other neurodegenerative diseases. The experimental
vector developed for delivery of corrected genes to our children is now being
used in clinical trials for Parkinson's disease.
We are a major contributor to the ongoing Gene Therapy Trial for Canavan Disease
currently at Cooper Health System, a division of Robert Wood Johnson Hospitals,
Camden, N.J. This trial is led by the world's leading researcher in gene therapy
for Canavan disease, Paola Leone, Ph.D.
We are also funding Evan Snyder, M.D., Ph.D.,
a leading researcher in the field of stem cells,
at Harvard Institutes of Medicine, as well as
Dr. Morris Baslow, who is also a renowned
expert in Canavan disease at The Center For Neurochemistry.
Dr. Baslow is collaborating with researchers at Kyoto University in Japan to
develop a non-invasive drug therapy to improve the lives of the children battling
Canavan disease.
Because Canavan serves as a model for other neurological diseases, our advancements
can also benefit millions suffering from debilitating neurological diseases such
as ALS, Parkinson's, Alzheimer's, Multiple Sclerosis, and stroke.
In addition to gene therapy, we are also funding groundbreaking research in the
field of stem cells. Canavan children may be among the first to benefit from
the great potential stem cells have to repair brain damage. We also support research
to develop a pharmacological approach towards halting the devastation of this
disease while researchers are racing for a cure. The benefits of our research
also offers hope to other neurological diseases and stroke.
Paola Leone, Ph.D.
Dr. Leone is the Director of the Cell & Gene Therapy Center and Associate
Professor in the Division of Neurosurgery at UMDNJ-Robert Wood Johnson Medical
School. Dr. Leone is also the Principal Investigator of the Canavan gene therapy
protocol using adeno-associated viral vectors. She is the leading researcher
in the field of gene therapy for Canavan disease and Dr. Leone was awarded the
UNESCO Lifetime Achievement Award for her devotion to saving the children. Dr.
Leone is an adjunct member of the Coriell Center for Medical Research.
Following post-doctoral studies in Montreal, she was an Associate Research Scientist
at Yale University from 1996-1998. From 1998-2001, she was Associate Director
of the CNS Gene Therapy Center at Jefferson Medical College and an Assistant
Professor in the Department of Neurosurgery. Dr. Leone has been responsible for
the development and characterization of viral (AAV, adenovirus, retrovirus) and
non-viral vectors for the treatment of Canavan Disease and other disorders. At
Yale and Thomas Jefferson Universities, she was the IND/FDA sponsor of two separate “Gene
Therapy for Canavan Disease” studies.
Current research interests of Dr. Leone's laboratory are also in in vivo studies
of viral vectors as well as stem cells and their use in neural transplantation
for therapeutic applications on a variety of neurodegenerative disorders, brain & spinal
injuries as well as stroke. The laboratory also works on pharmacological, genetic
and stem cell therapies of animal models of Canavan Disease, Amyotrophic Lateral
Sclerosis, Parkinson's Disease, Tay Sachs and other neurological disorders.
Evan Y. Snyder, M.D.,Ph.D.
in Neurobiology
Harvard Medical School
Boarded in Pediatrics, Pediatric Neurology, Neonatology.
Dr. Snyder is a world-renowned researcher and co-founder of the stem cell field.
His work focuses on understanding the mechanisms underlying Canavan disease and
other childhood neurodegenerative diseases. Dr. Snyder's research investigates
Canavan at a molecular and cellular basis--particularly as programmed into the
Central Nervous System at the stem cell level.
Dr. Snyder is studying the potential treatment of stem-cells in Canavan disease.
His work will focus on studying stem cells in Canavan animal models with the
goal of treating afflicted Canavan children.
Dr. Morris H. Baslow
Dr. Baslow is a renowned expert in Canavan disease and the study of N-acetyl
aspartate. He is currently at the Center For Neurochemistry, Orangeburg, New
York. Dr. Baslow is presently collaborating with researchers at Kyoto University
in Japan, to develop a non-invasive drug therapy for children with Canavan
disease. |
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with Paola Leone, Ph.D. Director
of the Cell & Gene Therapy Center in the Division
of Neurosurgery at UMDNJ-Robert Wood Johnson Medical
School |
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Evan
Y. Snyder,
Harvard
Medical School
Boarded in Pediatrics, Pediatric Neurology, Neonatology |
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