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Discovery Medicine / No 035


Gene Therapy for Hemophilia A

Abstract: Hemophilia A is an X-linked bleeding disorder caused by defective coagulation Factor VIII (FVIII). Although the efficacies of existing treatment using purified or recombinant FVIII are good, there remain shortcomings in using this particular form of treatment. A few FVIII gene therapy clinical trials have been initiated with modest improvements recorded, but these are no longer being continued due to insufficient efficacy. However, with the progress in the development of gene delivery vectors and the availability of mouse and canine hemophilia A models, gene therapy of hemophilia A remains an area of hot pursuit. ... Read more

"Dressed-up" Naked Plasmids: Emerging Vectors for Non-viral Gene Therapy

Abstract: The non-viral vectors and targeting methods offer some specific advantages. These include better safety profiles (lower toxicity and non-infectious properties) and the capacity to transfer large genes and low production costs. The clinical usefulness of non-viral methods has been hindered by their relatively low gene delivery and transgene expression efficiencies. However, recent problems in clinical trials using viral vectors renewed interest in non-viral technologies, particularly that the properties of non-viral vectors make them appear closer to the traditional pharmaceutics. ... Read more

Improving Anti-HCV Therapy

Abstract: Hepatitis C virus (HCV) infects about 2% of the world population or approximately 123 million people. The disease causes a significant level of morbidity and mortality among those afflicted. The combination of interferon and ribavirin is effective in many patients. However, the right treatment doses and duration, among other attributes, have a large impact on the treatment outcome. ... Read more

The Genetic and Environmental Basis of Hepatocellular Carcinoma

Abstract: Liver cancer is among the most common malignancies, impacting significantly across all societies worldwide. The lethal impact of this prevalent cancer is unlikely to change considerably in the near future due to a limited understanding of disease pathogenesis on the molecular, cellular, and environmental levels and how current knowledge might be converted into new preventive, therapeutic and diagnostic approaches. This article highlights the current challenges and opportunities in this critical area of unmet need. ... Read more

The Impact of Preventive HPV Vaccination

Abstract: The clearly defined causation by papillomavirus (HPV) and precursor lesions of cervical cancer have made this cancer largely preventable through Pap screening programs, and now by vaccination with Gardasil. This represents an important public health success story and here the authors summarize the potential impact of preventive HPV vaccination and some of the outstanding questions. ... Read more

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