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Page 1
Cone Health and Retinoids.
Kono M. Kono M. Prog Mol Biol Transl Sci. 2015;134:465-76. doi: 10.1016/bs.pmbts.2015.06.002. Epub 2015 Jul 2. Prog Mol Biol Transl Sci. 2015. PMID: 26310171 Review.
Gene therapy for RPE65-related retinal disease.
Miraldi Utz V, Coussa RG, Antaki F, Traboulsi EI. Miraldi Utz V, et al. Ophthalmic Genet. 2018 Dec;39(6):671-677. doi: 10.1080/13816810.2018.1533027. Epub 2018 Oct 18. Ophthalmic Genet. 2018. PMID: 30335549 Review.
The rapidly evolving state of gene therapy.
Gruntman AM, Flotte TR. Gruntman AM, et al. FASEB J. 2018 Apr;32(4):1733-1740. doi: 10.1096/fj.201700982R. FASEB J. 2018. PMID: 31282760 Review.
Food and Drug Administration has recently approved Luxturna (Spark Therapeutics Inc, Philadelphia, PA, USA), a recombinant adeno-associated virus (rAAV) 2 gene therapy for one type of Leber congenital amaurosis 2 ( 1 , 2 ). The European Medicines Agenc …
Food and Drug Administration has recently approved Luxturna (Spark Therapeutics Inc, Philadelphia, PA, USA), a recombinant adeno-associated …
[Gene Therapy for Inherited RETINAL AND OPTIC NERVE Disorders: Current Knowledge].
Ďuďáková Ľ, Kousal B, Kolářová H, Hlavatá L, Lišková P. Ďuďáková Ľ, et al. Cesk Slov Oftalmol. 2016 Fall;72(4):128-136. Cesk Slov Oftalmol. 2016. PMID: 27860478 Free article. Review. Czech.
At the time of writing this review gene-based clinical trials have been registered for Leber congenital amaurosis 2 (LCA2), retinitis pigmentosa 38, Usher syndrome 1B, Stargardt disease, choroideremia, achromatopsia, Leber hereditary optic neuropathy ( …
At the time of writing this review gene-based clinical trials have been registered for Leber congenital amaurosis 2