Ophthalmogenetik: Einblicke in aktuelle Methoden der Diagnostik, Phänotypisierung und Therapie

• Literatur

• 1 Cideciyan AV, Jacobson SG, Drack AV. et al. Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect. Nat Med 2019; 25: 225-228 doi:10.1038/s41591-018-0295-0
• 2 Vighi E, Trifunović D, Veiga-Crespo P. et al. Combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration. Proc Natl Acad Sci U S A 2018; 115: E2997-E3006 doi:10.1073/pnas.1718792115