1. (2017) Gene therapy clinical trials worldwide. Provided by the Journal of Gene Medicine. http://www.abedia.com/wiley/. Accessed 14 Apr 2020
2. (2018) NCT03655678: A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia. https://clinicaltrials.gov/ct2/show/NCT03655678. Accessed 14 Apr 2020
3. (2019) NCT03745287: A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease. https://clinicaltrials.gov/ct2/show/NCT03745287. Accessed 14 Apr 2020
4. (2019) NCT03872479: Single Ascending Dose Study in Participants With LCA10. https://clinicaltrials.gov/ct2/show/NCT03872479. Accessed 14 Apr 2020
5. Abudayyeh OO, Gootenberg JS, Essletzbichler P, Han S, Joung J, Belanto JJ, Verdine V, Cox DBT, Kellner MJ, Regev A, Lander ES, Voytas DF, Ting AY, Zhang F (2017) RNA targeting with CRISPR-Cas13. Nature 550:280–284. https://doi.org/10.1038/nature24049