Antisense-mediated inhibition of protein synthesis : rational drug design, pharmacokinetics, intracerebral application, and organ uptake of phosphorothioate oligodeoxynucleotides.

There is hardly any class of drugs for which the term "ratronal drug design" is more appropriate than for the currently developing antisense therapeutics. The specrficlty of the hybridization reaction and the surprisingly efficient uptake of synthetrc oligonucleotide derivatives provide a new class of selective protein synthesis inhibitors. Concurrently with the development of the antisense technology, elucidation of the pathogenetic role of mdrvrdual proteins for certain diseases is rapidly progressing, most notably in the fields of cancer research and virology. Consequently, the first clinical trtals are being conducted with antrsense therapeutics for the treatment of viral diseases and neoplasms.