BRAIN Publication-derived

Patient-Specific Retinal Organoids for RP Gene Therapy

Source Deng et al. · Wenzhou Medical University / National Eye Institute (NEI), China / USA · 10.1016/j.stemcr.2018.02.010

👤 Deng WL, Gao ML, Lei XL, Lv JN, Zhao H, He KW, Xia XX, Li LY, Chen YC, Li YP, Pan D, Xue T, Jin ZB ⏱ 72000 min 🧫 Patient-Derived iPSC (Retinitis Pigmentosa)

Abstract

Deng et al. established patient-derived iPSC retinal organoids from individuals with retinitis pigmentosa as a preclinical platform for screening AAV gene therapy vectors, demonstrating successful photoreceptor transduction and functional rescue of the RP phenotype.

Cell source

Patient-Derived iPSC (Retinitis Pigmentosa)

Application

Retinitis Pigmentosa Gene Therapy Development

Full SOP

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Attribution

This SOP was authored by Organthis based on the published method in Deng et al.. The originating laboratory holds no rights in this SOP and has not endorsed it unless marked Verified.

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