Format

Send to

Choose Destination
See comment in PubMed Commons below
Nat Rev Drug Discov. 2002 Jul;1(7):503-14.

Nucleic-acid therapeutics: basic principles and recent applications.

Author information

1
Department of Hematology, Pommeranian Academy of Medicine, Ul Rybacka 1, 71-252 Szczecin, Poland.

Abstract

The sequencing of the human genome and the elucidation of many molecular pathways that are important in disease have provided unprecedented opportunities for the development of new therapeutics. The types of molecule in development are increasingly varied, and include antisense oligonucleotides and ribozymes. Antisense technology and catalytic nucleic-acid enzymes are important tools for blocking the expression of abnormal genes. One FDA-approved antisense drug is already in the clinic for the treatment of cytomegalovirus retinitis, and other nucleic-acid therapies are undergoing clinical trials. This article reviews different strategies for modulating gene expression, and discusses the successes and problems that are associated with this type of therapy.

PMID:
12120257
DOI:
10.1038/nrd837
[Indexed for MEDLINE]
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Nature Publishing Group
    Loading ...
    Support Center