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Potential
targets – innumerable
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Promising
treatments – attainable
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Plenty
of Challenges – surmountable
The concept of gene therapy
has long appealed to biomedical researchers and
clinicians because of the promise to treat certain
diseases at their origin. However, negative
results and publicity have necessitated a low
profile for gene therapy research. Researchers
are, in fact, quietly making progress and are
confident that gene transfer will be elevated from
its current experimental status to a therapeutic
modality. Scientifically, major challenges
remain: targeting the right gene to the right
location in the right cells and expressing it at the
right time, all while minimizing any adverse
reactions. In addition, collaborations between
academia and industry are of critical importance for
the future of gene therapy. Substantial knowledge as
well as regulatory and financial resources is needed
to move gene therapy from small-scale endeavors to
clinical use. CHI’s Inaugural Targeting Gene
Therapy & Gene Transfer: Translation from Basic
Research to Clinical Results assembles prominent
scientific researchers, clinicians, businessmen, and
regulators who represent wide areas of expertise to
provide the forum for discussion for expanding the
potential and promise of gene therapies.
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