CRISPR/Cas9-mediated genome editing: From basic research to translational medicine

Area of Science:

• Biotechnology
• Genetics
• Molecular Biology

Background:

• The CRISPR/Cas9 system is a revolutionary programmable genome-editing tool.
• It enables precise creation of cellular and animal models for studying human diseases.
• CRISPR/Cas9 holds significant therapeutic potential, especially for gene therapy.

Purpose of the Study:

• To review in vitro, in vivo, and ex vivo applications of CRISPR/Cas9 in human disease research.
• To explore the potential of CRISPR/Cas9 in translational medicine.
• To discuss challenges hindering the clinical use of CRISPR/Cas9.

Main Methods:

• Literature review of CRISPR/Cas9 applications in disease research.
• Analysis of CRISPR/Cas9's role in developing disease models.
• Evaluation of CRISPR/Cas9's therapeutic potential and limitations.

Main Results:

• CRISPR/Cas9 facilitates precise genetic correction for treating genetic diseases.
• The technology aids in understanding genetic contributions to disease development.
• Significant progress has been made in various CRISPR/Cas9 applications.

Conclusions:

• CRISPR/Cas9 has revolutionized biological research and disease modeling.
• Clinical translation requires overcoming challenges in efficiency, specificity, and delivery.
• Further research is crucial for the safe and effective clinical application of CRISPR/Cas9.