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Gene Transfer Technology in Therapy: Current Applications and Future Goals

^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

Correspondence: Gaetano Romano, M.D., Kimmel Cancer Center, Jefferson Medical College, Thomas Jefferson University, 624 Bluemle Life Sciences Building, 233 South Street, Philadelphia, Pennsylvania 19107, USA. Telephone: 215-503-4511; Fax: 215-923-0249; e-mail: Gaetano.Romano{at}mail.tju.edu

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.

Key Words. Gene therapy • Clinical trials • Gene delivery systems in vivo or in vitro • Retroviruses • Adenovirus • Adeno-associated virus • Cationic liposomes

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