CRISPR Editing in Biological and Biomedical Investigation

Area of Science:

• Genomics
• Molecular Biology
• Biomedical Research

Background:

• The clustered regularly interspaced short palindromic repeat (CRISPR)-CRISPR-associated protein 9 (Cas9) system is a groundbreaking genome editing technology.
• CRISPR-Cas9 has significantly advanced biological and biomedical research, enabling the creation of animal models for human diseases.

Purpose of the Study:

• To review the latest advancements of the CRISPR-Cas9 system across various platforms.
• To discuss the challenges and limitations hindering the widespread application of CRISPR-Cas9 technology.
• To highlight the potential of CRISPR-Cas9 in disease mechanism elucidation and therapeutic development.

Main Methods:

• Review of current scientific literature on CRISPR-Cas9 applications.
• Analysis of different platforms utilizing CRISPR-Cas9 technology.
• Discussion of challenges in disease modeling, gene correction, and clinical translation.

Main Results:

• CRISPR-Cas9 facilitates the recapitulation of human diseases in diverse animal models.
• The system aids in understanding disease mechanisms and correcting causative mutations.
• Expanded applications include transcriptome and epigenome manipulation, with clinical potential.

Conclusions:

• CRISPR-Cas9 technology offers powerful tools for biological and biomedical research.
• Further development is needed to overcome existing hurdles for broader clinical use.
• The system holds significant promise for advancing human health and disease treatment.