Translatability barriers between preclinical and clinical trials of AAV gene therapy in inherited retinal diseases
Alizeh Shamshad, Chaerim Kang, Laura A. Jenny, Elijah M. Persad‐Paisley, Stephen H. Tsang
Topics & Concepts
Genetic enhancementGene deliveryBlindnessMedicineAdeno-associated virusClinical trialTransgeneRetinal degenerationNeuroscienceRetinalBioinformaticsVector (molecular biology)GenePathologyBiologyGeneticsOphthalmologyOptometryRecombinant DNARetinal Development and DisordersVirus-based gene therapy researchCRISPR and Genetic Engineering