Folia Pharmacologica Japonica
Online ISSN : 1347-8397
Print ISSN : 0015-5691
ISSN-L : 0015-5691
Antisense Strategy -Drug development for gene therapy-
Akira Murakami
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JOURNAL FREE ACCESS

1996 Volume 108 Issue supplement Pages 18-23

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Abstract
Translational inhibition by oligonucleotides complementary to mRNA leads us to the new field of the gene therapy. The concept, so called antisense strategy, has been applied to cure several incurable diseases such as HIV infection and cancer, and moreover to study functions of proteins and genes. Some of those trials are very promising and in the Phase II status. On the other hand, these days, several problems have been critically cited. They are mainly concerning with the mechanism of antisense effects. How do they penetrate into cells? How do antisense DNAs interact with target genes? How do they interact with endogenous proteins? Are there any non-antisense mechanism? Those problems must be solved before the strategy is to be used to patients. This review deals with the detailed evaluation of such problems and with the future prospects of the antisense strategy.
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© The Japanese PharmacologicalSociety
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