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Published Online: 8 July 2004

Progress in the Delivery of Therapeutic Oligonucleotides: Organ/Cellular Distribution and Targeted Delivery of Oligonucleotides In Vivo

Publication: Antisense and Nucleic Acid Drug Development
Volume 13, Issue Number 3


Oligonucleotide (ODN) therapy is a powerful tool for modulation of gene expression in vivo. With advances in ODN chemistry and progress in formulation development, ODNs are becoming widely acceptable drugs. This review summarizes the current status and future trend of the in vivo application of ODN therapeutics, especially antisense ODNs. Here, we review the current understanding of the tissue/organ distribution and cellular uptake of ODN drugs administered parenterally or nonparenterally to intact animals. The problems and advantages inherent in the use of different delivery methods for the treatment of particular diseases are discussed in detail. Emphasis is placed on the most widely studied ODN analogs, the phosphorothioates (PS). Lessons learned from antisense PS studies have broad implications for ODN therapeutics in general.

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cover image Antisense and Nucleic Acid Drug Development
Antisense and Nucleic Acid Drug Development
Volume 13Issue Number 3June 2003
Pages: 169 - 189
PubMed: 12954117


Published online: 8 July 2004
Published in print: June 2003


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Laixin Wang
Salus Therapeutics, Salt Lake City, UT 84108.
Ramesh K. Prakash
Salus Therapeutics, Salt Lake City, UT 84108.
C.A. Stein
Department of Oncology, Albert Einstein-Montefiore Cancer Center, Bronx, N.Y. 10467
Richard K. Koehn
Salus Therapeutics, Salt Lake City, UT 84108.
Duane E. Ruffner
Salus Therapeutics, Salt Lake City, UT 84108.

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