CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

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Science Advances  30 Oct 2019:
Vol. 5, no. 10, eaax1210
DOI: 10.1126/sciadv.aax1210

Article Information

vol. 5 no. 10

Online ISSN: 
  • Received for publication February 25, 2019
  • Accepted for publication September 13, 2019
  • .

Author Information

  1. Dong Hyun Jo1,*,
  2. Dong Woo Song2,*,
  3. Chang Sik Cho1,
  4. Un Gi Kim2,
  5. Kyu Jun Lee2,
  6. Kihwang Lee3,
  7. Sung Wook Park1,,
  8. Daesik Kim4,
  9. Jin Hyoung Kim1,
  10. Jin-Soo Kim4,
  11. Seokjoong Kim2,
  12. Jeong Hun Kim1,5,6, and
  13. Jung Min Lee2,7,
  1. 1Fight Against Angiogenesis-Related Blindness (FARB) Laboratory, Clinical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
  2. 2ToolGen Inc., Seoul, Republic of Korea.
  3. 3Department of Ophthalmology, Ajou University School of Medicine, Suwon, Republic of Korea.
  4. 4Center for Genome Engineering, Institute for Basic Science (IBS), Seoul, Republic of Korea.
  5. 5Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul, Republic of Korea.
  6. 6Department of Ophthalmology, Seoul National University College of Medicine, Seoul, Republic of Korea.
  7. 7School of Life Science, Handong Global University, Pohang 37554, Republic of Korea.
  1. Corresponding author. Email: steph25{at} (Je.H.K.); jmlee7706{at} (J.M.L.)
  • * These authors contributed equally to this work.

  • Present address: Seran Eye Center, Seoul, Republic of Korea.


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