What rheumatologists need to know about CRISPR/Cas9.

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• CRISPR/Cas9 genome editing technology emerged in 2012, rapidly advancing molecular biology.
• Initial concerns regarding specificity and off-target effects have been largely addressed by technological improvements.

Purpose of the Study:

• To review the applications of CRISPR/Cas9 for gene mutation creation.
• To discuss novel CRISPR/Cas9 adaptations for gene activation, repression, multiplexing, and screening.
• To explore the use of CRISPR/Cas9 in dissecting gene function, noncoding regions, and disease-related networks.

Main Methods:

• Review of CRISPR/Cas9 applications in eukaryotic systems.
• Analysis of advancements in CRISPR/Cas9 specificity and gene editing capabilities.
• Examination of diverse CRISPR/Cas9 adaptations for functional genomics and disease modeling.

Main Results:

• CRISPR/Cas9 enables precise gene mutation and the introduction of new sequences.
• New adaptations facilitate gene activation, repression, multiplexing, and screening for gene function.
• CRISPR/Cas9 is instrumental in dissecting gene networks, noncoding regions, and developing disease models.
• Therapeutic applications are advancing, with some entering human clinical trials.

Conclusions:

• CRISPR/Cas9 technology has evolved into a sophisticated tool for biological research.
• Its applications are expanding for understanding complex biological systems and disease mechanisms.
• CRISPR/Cas9 holds significant promise for developing novel therapeutics, including for rheumatic diseases.