2024
Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions
Banou L, Sarrafpour S, Teng C, Liu J. Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions. The Yale Journal Of Biology And Medicine 2024, 97: 491-503. PMID: 39703610, PMCID: PMC11650918, DOI: 10.59249/hwid7537.Peer-Reviewed Original ResearchConceptsAdeno-associated virusOcular gene therapyGene therapyFood and Drug AdministrationViral vectorsImmune responsePotential application of gene therapyClinical trialsApproval of voretigene neparvovecViral vector-based therapyApplication of gene therapyImproved delivery techniquesVector-based therapyImmune-privileged statusInduce immune responsesReduced immune responseVoretigene neparvovecContralateral eyeRetinal dystrophyImmune privilegeOcular diseasesDeliver genesEye diseaseTherapeutic efficacyTherapy
2017
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
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