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Delivery Approaches for Therapeutic Genome Editing and Challenges
Ilayda Ates1, Tanner Rathbone1, Callie Stuart1
1Department of Bioengineering, Clemson University, Clemson, SC 29634, USA.
Genes
|September 26, 2020
Summary
Gene editing technologies like CRISPR-Cas9 offer powerful therapeutic potential. However, challenges remain in safely and effectively delivering these gene-editing tools to target cells for treating diseases.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) have enabled therapeutic advances.
- Clustered regularly interspaced short palindromic repeats (CRISPR) and associated protein 9 (Cas9) offer enhanced gene-editing efficiency and tailorability.
Purpose of the Study:
- To review the challenges and considerations for improving gene editing therapies.
- To highlight issues related to the delivery of gene-editing tools into clinically relevant cells.
Main Methods:
- Literature review of gene-editing technologies and delivery methods.
- Analysis of current challenges in therapeutic gene editing.
Main Results:
- CRISPR-Cas9 provides unprecedented gene-editing capabilities for various diseases.
- A major barrier to therapeutic gene editing is the lack of safe and effective delivery methods for gene-editing reagents.
Conclusions:
- Improving the delivery of gene-editing tools is crucial for advancing therapeutic applications.
- Addressing delivery challenges will facilitate the clinical use of gene editing for inherited and acquired diseases.
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