Leber congenital amaurosis: Can CRISPR cure genetic blindness?

A clinical trial in the United States is looking to cure a type of genetic blindness called Leber congenital amaurosis (LCA) with the use of CRISPR technology.

For more information on this clinical trial please visit:
http://ir.editasmedicine.com/news-releases/news-release-details/allergan-and-editas-medicine-initiate-brilliance-phase-12

This video was made by McMaster Demystifying Medicine students: Iman Adani, Amanpreet Singh, Maham Tariq, Hibo Rijal and Vysh Satyajit.

Copyright McMaster University 2019.

References:

Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Trial of AGN-151587 (EDIT101) for the Treatment of LCA10. (n.d.). Retrieved November 7, 2019, from Editas Medicine
website: http://ir.editasmedicine.com/news-releases/news-release-details/allergan-and-editasmedicine-initiate-brilliance-phase-12

Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., … Ali, R. R.
(2008). Effect of Gene Therapy on Visual Function in Leber’s Congenital Amaurosis. New
England Journal of Medicine, 358(21), 2231–2239. https://doi.org/10.1056/NEJMoa0802268

Cideciyan, A. V., & Jacobson, S. G. (2019). Leber Congenital Amaurosis (LCA): Potential for
Improvement of Vision. Investigative Ophthalmology & Visual Science, 60(5), 1680–1695.
https://doi.org/10.1167/iovs.19-26672

Gogate, P., Kalua, K., & Courtright, P. (2009). Blindness in Childhood in Developing Countries: Time
for a Reassessment? PLoS Medicine, 6(12). https://doi.org/10.1371/journal.pmed.1000177

Ramirez, V. B. (2019, July 28). First Human CRISPR Trial in the US Aims to Cure Inherited
Blindness. Retrieved October 17, 2019, from Singularity Hub website:
https://singularityhub.com/2019/07/28/first-human-crispr-trial-in-the-us-aims-to-cure-inheritedblindness/

Single Ascending Dose Study in Participants With LCA10—Full Text View—ClinicalTrials.gov.
(n.d.). Retrieved November 7, 2019, from https://clinicaltrials.gov/ct2/show/NCT03872479

Weleber, R. G., Francis, P. J., Trzupek, K. M., & Beattie, C. (1993). Leber Congenital Amaurosis. In
M. P. Adam, H. H. Ardinger, R. A. Pagon, S. E. Wallace, L. J. Bean, K. Stephens, & A.
Amemiya (Eds.), GeneReviews®. Retrieved from
http://www.ncbi.nlm.nih.gov/books/NBK1298/

Видео Leber congenital amaurosis: Can CRISPR cure genetic blindness? канала Demystifying Medicine