Therapy works in a mouse model of LCA

A work has just been published on PLoS Medicine, announcing the incredible results obtained with intraocular gene therapy plus oral pharmacological treatment on mice affected with LCA caused by mutations in the Lrat gene.

The authors sound very hopeful about future therapy in humans, and they do not rule out the possibility of restoring sight in adults, at least in some forms of LCA.

The full text is available online free of charge at
http://medicine.plosjournals.org/perlserv/?request=get-document&doi=10.1371/journal.pmed.0020333

Do check it out! It’s a bit technical but the main messages sure come through. This is all very exciting.

Take care and stay tuned for more!