CRISPR-mediated genome editing and human diseases.

Liquan Cai1, Alfred L Fisher2,3, Haochu Huang4

  • 1Marshall Institute for Interdisciplinary Research (MIIR), Marshall University, Weisberg Engineering Complex, Marshall University, 1628 Third Avenue, Huntington, WV 25703, USA.

Genes & Diseases
|September 28, 2018
PubMed
Summary

CRISPR gene editing technology offers powerful tools for understanding gene function and treating diseases like Barth syndrome and cystic fibrosis. CRISPR/Cas9 enables precise DNA correction in various models, advancing biomedical research and therapeutic applications.

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