Related Experiment Video
Updated: May 15, 2025
10:44
In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing
Published on: May 5, 2023
1.3K
Gene regulation technologies for gene and cell therapy
Gabriel L Butterfield1, Samuel J Reisman2, Nahid Iglesias1
1Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.
Summary
Gene therapy innovations offer precise control over gene expression for treating genetic disorders. Next-generation technologies promise expanded applications, though delivery and safety challenges persist.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Gene therapy holds significant promise for treating genetic disorders and advancing regenerative medicine.
- Unregulated gene production can limit clinical utility due to complications.
- Current research focuses on controlled gene expression technologies.
Purpose of the Study:
- To review next-generation technologies for precise gene expression modulation in gene therapy.
- To highlight advancements enabling expanded reach and efficacy of gene therapies.
- To discuss potential applications in treating genetic and multifactorial diseases.
Main Methods:
- Review of emerging technologies such as epigenome editing, RNA editing, and synthetic genetic circuits.
- Analysis of advancements in tissue-specific promoters, inducible systems, and targeted delivery.
- Exploration of transcriptional regulatory network modulation and cell state-responsive systems.
Main Results:
- Next-generation technologies offer powerful control over cellular functions.
- Advanced systems demonstrate improved specificity and efficacy in clinical trials.
- Emerging tools include epigenome editing, antisense oligonucleotides, RNA editing, transcription factor-mediated reprogramming, and synthetic genetic circuits.
Conclusions:
- Ongoing integration of biological insights and engineering innovations is expanding gene therapy potential.
- Precise gene expression control is key to overcoming current limitations.
- Gene therapy offers hope for treating both rare genetic and complex multifactorial diseases.
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