Preclinical Safety Evaluation of a Recombinant AAV8 Vector for X-Linked Retinoschisis After Intravitreal Administration in Rabbits
Marangoni D, Wu Z, Wiley HE, Zeiss CJ, Vijayasarathy C, Zeng Y, Hiriyanna S, Bush RA, Wei LL, Colosi P, Sieving PA. Preclinical Safety Evaluation of a Recombinant AAV8 Vector for X-Linked Retinoschisis After Intravitreal Administration in Rabbits. Human Gene Therapy 2014, 25: 202-211. PMID: 25211193, PMCID: PMC4275775, DOI: 10.1089/humc.2014.067.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsDependovirusDNA, RecombinantEye ProteinsGenetic TherapyGenetic VectorsIntravitreal InjectionsRabbitsRetinoschisisConceptsIntravitreal administrationDose groupEye groupClinical trialsOcular inflammatory reactionRs1-KO miceHigh-dose groupPreclinical safety evaluationNew Zealand white rabbitsAdeno-associated viral vectorZealand white rabbitsIrreversible tissue damageNovel gene therapy approachVitreous infiltratesIntraocular inflammationOcular inflammationInflammatory infiltrateOphthalmological examinationEye doseInflammatory cellsIntravitreal routeOcular safetyClinical signsCommon causeHistopathological analysis