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| FUND RESEARCH FOR NEUROMUSCULAR DISEASE..... (more to come soon....) |
DREAMS
The Phoenix Fox Research Fellowship was established
January 8th 2007. Its sole purpose is to raise money to
help fund research for a multitude of neuromuscular
diseases. Our primary recipient is Dr. Barry Byrne at the
University of Florida. Dr. Byrne is a brilliant Molecular
biologist / Pediatric Cardiologist who has been working on
Pompe since the early 90's . In particular, he has been
working on Gene therapy to treat different diseases but
mostly Pompe disease. The Powell Gene therapy center at
UF is his home base and primary place of operation. While
there have been a few variables in the Gene Therapy
approach over the years the primary principal has
remained largely the same. The goal is to use a type of
virus that has had its DNA payload modified to match the
missing or defective DNA of the Pompe patient. The virus
is then inserted into the body through I.V. or other means.
Then, using the mechanisms that have given viruses the
ability to traverse the human body for millions of years,
these viruses are able to get inside the cell and deposit the
modified DNA without harm to the cell or the existing DNA
in the cell. This "new" DNA is then read by the cell's
protein building system and begins to manufacture the
enzymes that Phoenix and so many other people are
missing. We are currently waiting for a clinical trial to test
this approach in humans. Hopefully this should take place
sometime in the next year......
January 8th 2007. Its sole purpose is to raise money to
help fund research for a multitude of neuromuscular
diseases. Our primary recipient is Dr. Barry Byrne at the
University of Florida. Dr. Byrne is a brilliant Molecular
biologist / Pediatric Cardiologist who has been working on
Pompe since the early 90's . In particular, he has been
working on Gene therapy to treat different diseases but
mostly Pompe disease. The Powell Gene therapy center at
UF is his home base and primary place of operation. While
there have been a few variables in the Gene Therapy
approach over the years the primary principal has
remained largely the same. The goal is to use a type of
virus that has had its DNA payload modified to match the
missing or defective DNA of the Pompe patient. The virus
is then inserted into the body through I.V. or other means.
Then, using the mechanisms that have given viruses the
ability to traverse the human body for millions of years,
these viruses are able to get inside the cell and deposit the
modified DNA without harm to the cell or the existing DNA
in the cell. This "new" DNA is then read by the cell's
protein building system and begins to manufacture the
enzymes that Phoenix and so many other people are
missing. We are currently waiting for a clinical trial to test
this approach in humans. Hopefully this should take place
sometime in the next year......
To help fund research for Phoenix and other children with neuromuscular
disease go to The University of Florida Foundation and specify Fox
Research Fellowship (013653).
disease go to The University of Florida Foundation and specify Fox
Research Fellowship (013653).
Molecular Genetics and Gene Therapy of Cardiovascular Disease
The laboratory is actively involved in developing new genetic
therapies for cardiovascular disease. In the area of cardiomyopathy,
we are studying gene replacement in an autosomal recessive form of
fatal cardiomyopathy in children. The disease is the prototype of
lysosomal storage disorders leading to skeletal and cardiac muscle
weakness. We have used AAV vectors to achieve sustained correction
of the gene deficiency and correction of the phenotype in natural and
transgenic mouse models of the disease. The current therapy is
currently being proposed for human clinical trials. Similar therapies
are being used to combat cardiac transplantation rejection. Secondly,
we are investigating the ability of mescnchymal stem cells to undergo
myocardial specification for the purpose of tissue repair in the heart.
Finally, several projects are focused on the use of AAV vectors
injected into striated muscle to achieve sustained release of
therapeutic proteins, including thrombolytic factors and coagulation
factors.
Barry J. Byrne
Professor
Postdoctoral Fellow, Johns Hopkins University
Intern and Resident, Johns Hopkins Hospital
M.D., University of Illinois
Ph.D., University of Illinois
B.S., Denison University
citations
Awards, Professional Service:
James Scholar, University of Illinois
Clinician Scientist Award, Johns Hopkins University
Gene Therapy - Reviewer
American Heart Association, Grant Review Panel
NIH: SBIR Study Section (1997, 1998)
NIH: NHLBI Study Section
Director, Powell Gene Therapy Center
Teaching Responsibilities:
MD/PhD Program Director
BMS 5003 Medical Aspects of Genetics
GMS 7191 Research Conference
GMS 7979 Advanced Research
GMS 7980 Doctoral Research
The laboratory is actively involved in developing new genetic
therapies for cardiovascular disease. In the area of cardiomyopathy,
we are studying gene replacement in an autosomal recessive form of
fatal cardiomyopathy in children. The disease is the prototype of
lysosomal storage disorders leading to skeletal and cardiac muscle
weakness. We have used AAV vectors to achieve sustained correction
of the gene deficiency and correction of the phenotype in natural and
transgenic mouse models of the disease. The current therapy is
currently being proposed for human clinical trials. Similar therapies
are being used to combat cardiac transplantation rejection. Secondly,
we are investigating the ability of mescnchymal stem cells to undergo
myocardial specification for the purpose of tissue repair in the heart.
Finally, several projects are focused on the use of AAV vectors
injected into striated muscle to achieve sustained release of
therapeutic proteins, including thrombolytic factors and coagulation
factors.
Barry J. Byrne
Professor
Postdoctoral Fellow, Johns Hopkins University
Intern and Resident, Johns Hopkins Hospital
M.D., University of Illinois
Ph.D., University of Illinois
B.S., Denison University
citations
Awards, Professional Service:
James Scholar, University of Illinois
Clinician Scientist Award, Johns Hopkins University
Gene Therapy - Reviewer
American Heart Association, Grant Review Panel
NIH: SBIR Study Section (1997, 1998)
NIH: NHLBI Study Section
Director, Powell Gene Therapy Center
Teaching Responsibilities:
MD/PhD Program Director
BMS 5003 Medical Aspects of Genetics
GMS 7191 Research Conference
GMS 7979 Advanced Research
GMS 7980 Doctoral Research
