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The new frontier: gene and oligonucleotide therapy
1Center for Lung Research, Vanderbilt University School of Medicine, Nashville, TN 37232-2650.
Pharmaceutica Acta Helvetiae
|January 1, 1994
Summary
Gene and oligonucleotide therapies offer new treatments for genetic, cancer, and infectious diseases. Pharmaceutical sciences are key to developing effective gene delivery systems and drug formulations for these advanced therapies.
Area of Science:
- Biotechnology
- Molecular Biology
- Pharmaceutical Sciences
Background:
- Gene and oligonucleotide therapies are emerging as clinically viable treatments.
- These therapies target genetic, neoplastic, and infectious diseases.
Purpose of the Study:
- To review current gene and oligonucleotide therapy approaches.
- To highlight the role of pharmaceutical sciences in advancing these therapies.
Main Methods:
- Gene therapy utilizes viral vectors (retrovirus, adenovirus, AAV, HSV-1) or bacterial plasmids for gene delivery.
- Plasmid DNA delivery employs methods like lipofection, polylysine conjugates, and artificial viral envelopes.
- Oligonucleotide therapy uses triple-helix-forming or antisense oligomers to block gene transcription or translation.
Main Results:
- Key applications include cystic fibrosis transmembrane regulator and alpha 1-antitrypsin gene replacement.
- Therapeutic strategies aim to arrest human immunodeficiency virus infection.
- Reversal of tumorigenicity and cancer immunization are significant areas of focus.
Conclusions:
- Gene and oligonucleotide therapies hold significant clinical promise for various diseases.
- Advancements in pharmaceutical chemistry, drug delivery, analytical chemistry, and biopharmaceutics are crucial for successful implementation.