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a Kimmel Cancer Center, Jefferson Medical
College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA;
b Department of Pathology, Anatomy and Cell
Biology, Jefferson Medical College, Thomas Jefferson University, and
Sbarro Institute for Cancer Research and Molecular Medicine,
Philadelphia, Pennsylvania, USA
Key Words. Gene therapy • Clinical trials • Gene delivery systems in vivo or in vitro • Retroviruses • Adenovirus • Adeno-associated virus • Cationic liposomes
Dr. Gaetano Romano, Kimmel Cancer Center, Jefferson Medical College, Thomas Jefferson University, 624 Bluemle Life Sciences Building, 233 South Street, Philadelphia, Pennsylvania 19107, USA.
Gene therapy has attracted much interest since the
first submissions of phase I clinical trials in the early 1990s, for
the treatment of inherited genetic diseases. Preliminary results were
very encouraging and prompted many investigators to submit protocols
for phase I and phase II clinical trials for the treatment of
inherited genetic diseases and cancer. The possible application of
gene transfer technology to treat AIDS, cardiopathies, and neurologic
diseases is under evaluation. Some viral vectors have already been
used to deliver HIV-1 subunits to immunize volunteers who are
participating in the AIDS vaccine programs in the USA. However, gene
delivery systems still need to be optimized in order to achieve
effective therapeutic interventions. The purpose of this review is to
summarize the latest achievements in improving gene delivery systems,
their current application in preclinical studies and in therapy, and
the most pressing issues that must be addressed in the area of vector
design. Stem Cells 1999;17:191-202
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