CRISPR/Cas9 in perspective: evaluating efficacy, delivery methods, and ethical challenges in genome editing

Area of Science:

• Biotechnology
• Genetics
• Molecular Biology

Background:

• DNA sequencing advances reveal disease-linked genes and genetic composition.
• Genetic engineering capabilities have been transformed by new technologies.
• Clustered regularly interspaced short palindromic repeats (CRISPR) associated protein 9 (Cas9) is a key innovation.

Purpose of the Study:

• Critically evaluate the CRISPR/Cas9 system.
• Examine its molecular mechanism, efficiency, delivery, and potential for unintended mutations.
• Discuss advancements and ethical implications of genome editing.

Main Methods:

• Review of CRISPR/Cas9 system.
• Comparative analysis with other gene-editing tools.
• Discussion of base editing, prime editing, and high-fidelity Cas variants.

Main Results:

• CRISPR/Cas9 is a powerful and precise genome editing tool.
• Applications include immunotherapy, agriculture, and human therapeutics.
• Concerns include off-target effects, delivery, safety, and ethics.

Conclusions:

• CRISPR/Cas9 presents significant potential and limitations.
• Advancements like base and prime editing are expanding capabilities.
• Future research should focus on responsible application and addressing ethical concerns.