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Antisense therapeutics: from theory to clinical practice.
Kathleen F Pirollo1, Antonina Rait, Leanne S Sleer
1Department of Oncology, Georgetown University Medical Center, Washington, DC 20007, USA.
Pharmacology & Therapeutics
|June 14, 2003
Summary
Antisense (AS) oligonucleotides show therapeutic potential, with many now advancing in clinical trials for cancer treatment. Recent advances in understanding their structure and chemistry are driving this progress.
Area of Science:
- Biochemistry
- Molecular Biology
- Pharmacology
Background:
- Antisense (AS) oligonucleotide technology was developed in the late 20th century.
- Significant advancements in understanding AS molecule structure and chemistry have occurred recently.
- The therapeutic potential of AS oligonucleotides has been recognized for decades.
Purpose of the Study:
- To review prominent modifications of AS oligonucleotides for clinical applicability.
- To focus on the clinical applications of AS oligonucleotides, particularly in cancer treatment.
- To detail the current status of AS clinical trials and identify future molecular targets.
Main Methods:
- Review of current scientific literature on AS oligonucleotide modifications.
- Analysis of clinical trial data for AS molecules.
- Identification of emerging AS molecules for future therapeutic development.
Main Results:
- A rapid increase in AS molecules progressing beyond Phase I clinical trials.
- Detailed description of AS oligonucleotide modifications relevant to clinical use.
- Identification of key targets for upcoming AS oligonucleotide therapies in oncology.
Conclusions:
- AS oligonucleotides are increasingly viable therapeutic agents, especially for cancer.
- Continued research into AS molecule chemistry and structure is crucial for clinical success.
- The pipeline for AS oligonucleotide-based cancer therapies is expanding rapidly.