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Drug Discov Today. 2001 Mar 1;6(6):303-315.

The cellular delivery of antisense oligonucleotides and ribozymes.

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Aston Centre for Gene-based Therapeutics (ACGT), Pharmaceutical Sciences Research Institute, Aston University, Aston Triangle, B4 7ET, Birmingham, UK


The design and development of antisense oligonucleotides and ribozymes for the treatment of diseases arising from genetic abnormalities has become a real possibility over the past few years. Improvements in oligonucleotide chemistry have led to the synthesis of nucleic acids that are relatively stable in the biological milieu. However, advances in cellular targeting and intracellular delivery will probably lead to more widespread clinical applications. This review looks at recent advances in the in vitro and in vivo delivery of antisense oligodeoxynucleotides and ribozymes.

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