Next-generation CRISPR technology for genome, epigenome and mitochondrial editing

Area of Science:

• Biotechnology
• Molecular Biology
• Genetics

Background:

• CRISPR technology is rapidly evolving, offering new tools for biomedical research.
• Existing CRISPR toolboxes and methods have significant potential to revolutionize scientific discovery and therapeutic approaches.

Purpose of the Study:

• To provide an updated overview of CRISPR toolboxes and methods.
• To critically evaluate the potential and challenges of CRISPR-based genome, epigenome, and mitochondrial editing.
• To discuss the current status of CRISPR/Cas9 clinical trials and future gene therapy perspectives.

Main Methods:

• Review and critical discussion of current CRISPR technologies.
• Analysis of technical challenges and solutions for in vivo epigenome editing, base editing, prime editing, and mitochondrial editing.
• Examination of CRISPR-associated transposases and integrases for large DNA integration.
• Assessment of CRISPR/Cas9 clinical trial data and ethical considerations.

Main Results:

• CRISPR offers diverse applications including nuclear, epigenome, and mitochondrial editing.
• Technical hurdles exist for in vivo applications, large DNA integration, and complex tissue editing, with ongoing solutions.
• CRISPR/Cas9 clinical trials show promise, but ethical and societal issues require careful consideration.

Conclusions:

• CRISPR technology holds immense promise for biomedical research and gene therapy.
• Addressing technical challenges and ethical concerns is crucial for the responsible advancement of CRISPR applications.
• Further research and clinical trials are essential to fully realize the therapeutic potential of CRISPR.