Novel therapeutics for Stargardt disease
Lu LJ, Liu J, Adelman RA. Novel therapeutics for Stargardt disease. Graefe's Archive For Clinical And Experimental Ophthalmology 2017, 255: 1057-1062. PMID: 28285324, DOI: 10.1007/s00417-017-3619-8.Peer-Reviewed Original ResearchConceptsStargardt diseaseVision lossABCA4 geneStem cell transplantationSevere vision lossPromising treatment candidateNovel therapeutic strategiesRetinal pigment epitheliumStem cell therapyVisual acuityStandard treatmentMacular degenerationTreatment candidatesHESC-RPE cellsStargardt patientsTherapeutic strategiesPharmacological agentsPigment epitheliumMacular dystrophyNovel therapeuticsPatientsDiseaseVisual cyclePrevalent formSecond decade