Litcius/Paper detail

Treatment-Emergent Adverse Events in Gene Therapy Trials for Inherited Retinal Diseases: A Narrative Review

Yan Nuzbrokh, Alexis Kassotis, Sara D. Ragi, Ruben Jauregui, Stephen H. Tsang

2020Ophthalmology and Therapy28 citationsDOIOpen Access PDF

Abstract

Patient safety is a primary priority in the conduction of retinal gene therapy trials. An understanding of risk factors and mitigation strategies for post-procedure complications is crucial for the optimization of gene therapy clinical trial protocols. In this review, we synthesize the literature on ocular delivery methods, vector platforms, and treatment-emergent adverse effects in recent gene therapy clinical trials for inherited retinal diseases.

Topics & Concepts

Genetic enhancementClinical trialMedicineAdverse effectRetinalNarrative reviewGene deliveryIntensive care medicineBioinformaticsGenePathologyOphthalmologyPharmacologyGeneticsBiologyRetinal Development and DisordersCRISPR and Genetic EngineeringRetinal and Macular Surgery