CRISPR/Cas9 therapeutics: progress and prospects

Area of Science:

• Biotechnology
• Genetics
• Molecular Biology

Background:

• CRISPR/Cas9 gene editing technology shows great promise for permanent disease correction and gene disruption.
• Development of efficient Cas9 variants addresses complex genomic alterations in diseases.
• Current limitations exist in the effective in vivo delivery of CRISPR systems to target cells.

Purpose of the Study:

• To review current developments in CRISPR/Cas9 gene editing for disease treatment.
• To examine gene-editing types, delivery vectors, and disease characteristics relevant to CRISPR applications.
• To summarize clinical trial successes and identify challenges in CRISPR/Cas9 targeted delivery.

Main Methods:

• Review of CRISPR/Cas9 gene-editing technologies and their variants.
• Analysis of nonviral delivery vectors with target recognition capabilities.
• Examination of disease-specific factors for targeted delivery and gene editing strategies.

Main Results:

• CRISPR/Cas9 technology enables precise correction of mutations and disruption of disease genes.
• Nonviral vectors with targeting functions are a focus for future in vivo delivery research.
• Disease characteristics can inform targeted delivery and gene editing approaches.

Conclusions:

• Effective in vivo delivery remains a significant hurdle for CRISPR/Cas9-based disease therapies.
• Tailoring gene-editing methods and delivery vectors to specific diseases is crucial.
• Further research is needed to overcome challenges in targeted delivery and clinical application of CRISPR/Cas9.