Li, Y., Kang, X.J., Pang, J.K.S. et al. Human germline editing: Insights to future clinical treatment of diseases. Protein Cell 10, 470–475 (2019). https://doi.org/10.1007/s13238-018-0594-8
Abstract:
Last year, the first attempt to genetically modify human embryos in the United States was reported and sparked a huge debate (Ma et al., 2017). Although the first human germline modification was only performed two years ago, the study showed that rapid advances in technology has allowed the rate of off-target effects and mosaicism to be reduced considerably (Liang et al., 2015). Recently, Vertex and CRISPR therapeutics collaborated and developed CTX001, the first CRISPR/Cas9-based therapy, targeting patients with β-thalassemia and have begun phase 1/2 clinical trials. With policies and technologies regarding genome editing both developing rapidly, explorations into the possibility of clinical gene editing for hundreds of hereditary diseases are starting to become achievable. Here, we address the progress of human embryo editing technologies so far and its promise and risks in advancing therapy for hereditary diseases.
License type:
http://creativecommons.org/licenses/by/4.0/
Funding Info:
This work was supported by the National Key R&D Program of China (2016YFC1000601), National Natural Science Foundation of China (Grant Nos. 81570101, 81728002, 81741006 and 81871162), Guangdong Province Science and Technology Project (2017A020214005) and the Guangzhou Science and Technology Project (201803010048). The authors apologize for the omission of additional applications of CRISPR/Cas9 or citations due to the reference limitations.