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• LCA / RPE65 gene defect
• Posted: 2009-03-03 16:50:43 By S B
• There's a natural source of 9-cis-retinal (which can replace the absent 11-cis-retinal caused by the RPE64 defect) available. Please follow this link to the RNIB forums, where the latest versions reside: http://www.rnib.org.uk/messageboard/thread.php?threadID=6243&topicSort=desc
  
  There's also trials under way on a synthetic retinoid which is supposed to do the same thing:
  
  Oral Retinoid Enters Trials
  A synthetic retinoid has begun a Phase 1 safety study. The orally administered compound is designed to replace 11-cis-retinal, a part of
  the retinoid-rhodopsin cycle that is essential for visual functioning in Lebers Congenital Amaurosis sufferers.
  The hypothesis of synthetic retinoid replacement therapy has been proved to be sound in earlier trials in rodent models.
  The open-label single centre study will assess the safety and tolerability of the compound when administered multiple times to healthy adult
  volunteers. The trial will examine six separate dose strengths. QLT hopes to announce preliminary data from the trial in the first quarter of 2009.
  Ophthalmology Times Europe
  October 2008
  
  Good luck!