Targeted genome editing tools for disease modeling and gene therapy

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• Custom-designed nucleases (CDNs), including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), have advanced genetic engineering.
• The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system represents a significant advancement in genome editing.

Purpose of the Study:

• To review progress in genome editing technologies: CDNs (ZFNs, TALENs) and CRISPR-Cas.
• To compare the key features and applications of these genome editing tools.
• To discuss future prospects in disease modeling and gene therapy.

Main Methods:

• Review of scientific literature on ZFNs, TALENs, and CRISPR-Cas systems.
• Comparative analysis of the efficiency, flexibility, and applications of each technology.
• Summary of current and potential uses in biological research and medicine.

Main Results:

• ZFNs and TALENs enable precise genetic engineering across various cell types and species.
• CRISPR-Cas systems demonstrate superior efficiency and flexibility compared to CDNs.
• All three technologies are applicable to modifying cells and model organisms.

Conclusions:

• CRISPR-Cas technology holds significant potential for biological studies and medical applications.
• Genome editing tools are crucial for advancing disease modeling and gene therapy.
• The field of genome editing is rapidly evolving with promising future directions.