PubMed · 11298852
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G Gahrton. 2001. Introduction.. https://doi.org/10.1046/j.1365-2796.2001.00804.x
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A key factor in the success of gene therapy is the development of gene delivery systems that are capable of efficient gene transfer in a broad variety of tissues, without causing any pathogenic effect. Currently, viral vectors based on many different viruses have been developed, and their performance and pathogenicity has been evaluated in animal models. The results of these studies form the basis for the first clinical trials for correcting genetic disorders using retroviral, adenoviral, and adeno-associated viral vectors. Even though the results of these trials are encouraging, vector development is still required to improve and refine future treatment of hereditary disorders.
As currently available preventive and therapeutic interventions for hypercholesterolemia are ineffective in a substantial proportion of patients, severe dyslipidemias associated with atherosclerotic vascular disease remain an important target for the development of novel gene therapies. The development of a safe and efficient gene transfer vector has been a major challenge in liver-directed gene therapy, but recently significant progress has been made in this area. Proof-of-principle experiments indicate that the transfer of lipid-modifying genes to the liver is an effective method to restore normal plasma lipids and protect against atherosclerosis. This article summarizes recent developments in liver-directed gene delivery and reviews data on the treatment of dyslipidemias and prevention of atherosclerosis in animals. The evidence presented suggests that some of the approaches taken in animals may be ready for clinical trials in the near future.
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