 Great
fanfare greeted the emergence of gene therapy into clinical medicine
nearly a decade ago. By using the potent tools of molecular biology
scientists seemed confident that they could soon cure a number of
diseases through insertion of healthy genes into patients. However,
within a few years gene therapy quickly fell out of favor the victim
of unreliable gene-delivery systems.
Indeed, splicing therapeutic genes
into the nuclei of target cells turned out to be both complex and
frustrating. A federal review panel criticized scientists for rushing
into clinical trials and urged a return to basic research that could
guarantee the long-term success of gene based therapies. Several
scientists at the University of Florida have been working for years
to use a tiny, harmless virus called adeno-associated virus (AAV)
as a delivery vehicle or vector for healthy genes.
AAV gene carriers were not used
in the first wave of gene-transfer experiments due to early laboratory
limitations. Those early limitations have recently been overcome
and many leaders in the field now believe that AAV may prove to
be a superior vector because it infects cells without detectable
side effects and it induces a very low immune response. As a result,
genes introduced by AAV can provide continuous production of recombinant
transgene following only a single application of therapeutic vector.
The primary goal of our research
program is to test the feasibility of AAV to deliver genes capable
of improving the clinical success of two transplantation procedures
amenable to persons with type 1 diabetes, as well as interrupting
one of the most debilitating complications of the diabetes retinopathy.
Mark Atkinson, Center Director
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Administration: Mark Atkinson
and Camillo Ricordi
Vector Production: Richard
Synder
Immunology and Pathology:
James Crawford
Islet Isolation and Functional Assessment: Camillo Ricordi
Transplantation: Luca Inverardi
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