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The Thrill of Victory the Agony of Defeat, the Trials of Developing
a New Class of Therapeutics: From Gene to Cell Therapy, A Case Study
Cori Gorman, PhD, MBA
Lecture at Annual Sigma Xi Meeting
El Paso, Texas
May 8, 2009
http://www.sigmaxi.org/chapters/lists/chapters.php
When the concept of gene therapy was conceived in the 1980s, bone marrow
transplantation had already been in clinical practice for decades, and
there was a spirited debate among investigators as to whether targeted
genetic disorders would first be corrected by gene therapy or cell
therapy. Looking back, it is apparent that the debate was based more on
individual preferences than on science, because from the very beginning
there were two main strategic means to achieve therapeutic gene transfer
in animals and humans: in vivo or ex vivo. “In vivo gene therapy” was
typically used in reference to the direct delivery of genes into diseased
organisms so as to achieve a therapeutic outcome. In contrast, “ex vivo
gene therapy” usually referred to the delivery of therapeutic genes into
cultured cells in vitro, followed by transplantation of the gene-corrected
cells into the diseased hosts.
The exciting, recent development of vector-mediated genetic reprogramming
of terminally differentiated adult cells into induced pluripotent stem
(iPS) cells, which exhibit many characteristics that imply they may be an
excellent alternative to ES cells for cell-based therapies and
regenerative medicine. Engineered tissues derived from iPS cells obtained
from patients might be autologously transplanted back into the same
patients without the need for lifelong immunosuppression; this would have
distinct advantages over the allogeneic transplantation procedures
currently in use.
Sigma Xi is an international, multidisciplinary research society whose
programs and activities promote the health of the scientific enterprise
and honor scientific achievement. There are nearly 60,000 Sigma Xi members
in more than 100 countries around the world. Sigma Xi chapters, more than
500 in all, can be found at colleges and universities, industrial research
centers and government laboratories. The Society endeavors to encourage
support of original work across the spectrum of science and technology and
to promote an appreciation within society at large for the role research
has played in human progress.
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