| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 6200434 | Ophthalmology | 2016 | 13 Pages |
Abstract
No serious safety problems were observed in the first 5 participants enrolled in this phase I trial of virus-based gene transfer in this mitochondrial disorder. Additional study follow-up of these and additional participants planned for the next 4 years is needed to confirm these preliminary observations.
Keywords
ETDRSscAAVPERGNaBLHONRNFLiOpAAVACFDMEMMitochondrial DNADulbecco's modified Eagle's mediumNeutralizing antibodyPattern electroretinogramearly treatment diabetic retinopathy studymtDNAIntraocular pressureRetinal nerve fiber layerLeber hereditary optic neuropathypolymerase chain reactionPCRAdeno-associated virus vector
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Authors
William J. MS, Joyce C. MS, Janet L. MD, Vittorio DSc, Phillip CCRP, Rajeshwari D. PhD, Huijun PhD, Anil MS, Byron L. MD, John MD,