CRISPR/Cas9-mediated genome editing in nonhuman primates.

Area of Science:

• Genetics and Genomics
• Translational Medicine
• Primate Models

Background:

• Non-human primates (NHPs) closely resemble humans, making them ideal models for studying complex human diseases.
• The development of genetic engineering tools has enabled the creation of more accurate disease models.

Purpose of the Study:

• To review the historical development and current progress of transgenic NHP models.
• To evaluate the application and limitations of CRISPR/Cas9 genome editing in NHP research.
• To discuss the future prospects of NHP models in human disease studies.

Main Methods:

• Review of existing literature on transgenic NHP models and CRISPR/Cas9 genome editing techniques.
• Analysis of advancements from initial proof-of-principle studies to complex disease models.
• Discussion of technical challenges, including off-target mutations and mosaicism.

Main Results:

• Successful generation of transgenic NHPs, including models for human neurodegenerative diseases.
• CRISPR/Cas9 technology has significantly advanced NHP model creation, enabling precise genetic modifications.
• Sophisticated NHP models now accurately phenocopy complex human disease features.

Conclusions:

• CRISPR/Cas9-mediated genome editing offers significant advantages for creating NHP disease models.
• Addressing concerns such as off-target and mosaic mutations is crucial for reliable NHP modeling.
• Ongoing improvements in genome editing techniques promise enhanced prospects for human disease research using NHPs.