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Updated: Jul 8, 2026
10:18
In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium
Published on: May 6, 2018
[Prospects of gene therapy]
Małgorzata Barańska1, Jadwiga Skretkowicz
1Z Zakładu Farmakogenetyki Uniwersytetu Medycznego w Lodzi.
Wiadomosci Lekarskie (Warsaw, Poland : 1960)
|January 8, 2008
Summary
Gene therapy offers two main strategies: repairing damaged genes or modifying gene expression to treat diseases at a molecular level. While clinical trials are increasing for cancer and genetic disorders, safe and effective gene therapy remains a research challenge.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Context:
- Gene therapy has seen extensive development, with two primary research approaches.
- Clinical trials for gene therapy are rapidly expanding.
- Significant methodological progress has been made, but challenges remain.
Purpose:
- To explore gene therapy strategies for treating diseases at the molecular level.
- To discuss the use of gene therapy for cancer, monogenic, and cardiovascular diseases.
- To highlight the potential of gene therapy for autoimmune diseases and RNA interference (RNAi).
Summary:
- Gene therapy involves replacing damaged genes in somatic cells or modifying gene expression to inhibit pathogenic protein biosynthesis.
- Retroviral, adenoviral vectors, and plasmid DNA are commonly used for gene delivery.
- RNA interference (RNAi) using small interfering RNA (siRNA) is a newer approach to inhibit gene expression.
Impact:
- Gene therapy offers novel treatment modalities for various diseases, including genetic disorders and cancer.
- The development of effective gene delivery vectors is crucial for successful gene therapy.
- Further research is needed to overcome challenges and ensure the safety and efficacy of gene therapy.
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