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Generation of a monkey with MECP2 mutations by TALEN-based gene targeting
1Institute of Neuroscience, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, 200031, China.
Neuroscience Bulletin
|May 20, 2014
Summary
Researchers generated a cynomolgus monkey with MECP2 mutations using TALENs gene editing. The mutation led to the monkey
Area of Science:
- Neuroscience
- Genetics
- Developmental Biology
Background:
- Gene editing in model organisms advances understanding of brain development and diseases.
- MECP2 gene is critical for neural development.
Purpose of the Study:
- To generate a cynomolgus monkey model with MECP2 mutations using TALENs-mediated gene targeting.
- To assess the feasibility of site-specific gene editing in non-human primates.
Main Methods:
- TALENs mRNA was injected into in vitro fertilized monkey zygotes.
- Embryos were transplanted into surrogate monkeys.
- Gene editing was confirmed in the resulting offspring.
Main Results:
- One male newborn cynomolgus monkey with an MECP2 deletion (frameshifting mutation) was generated.
- The mutation resulted in near-complete depletion of MeCP2 protein in the monkey's brain.
- The affected monkey did not survive post-birth.
Conclusions:
- Site-specific gene editing is feasible in non-human primates like cynomolgus monkeys.
- MECP2 is essential for survival, highlighting its critical role in neural development.
- This model offers new avenues for studying MECP2-related disorders.
