학술논문
AAV‐mediated gene therapy improving mitochondrial function provides benefit in age‐related macular degeneration models.
Document Type
Article
Author
Source
Subject
*MACULAR degeneration
*PHOTORECEPTORS
*GENE therapy
*MITOCHONDRIA
*AEROBIC capacity
*COMPLEMENT factor H
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Language
ISSN
2001-1326
Abstract
Twenty-eight-hour post-transduction transduced cells and a minimum of 16 wells of untransduced cells were insulted with 5-mM NaIO3 and 12 h post-insult cells underwent a mitochondrial stress test using an XFe96 Seahorse. AAV-mediated gene therapy improving mitochondrial function provides benefit in age-related macular degeneration models AAV2/8-ophNdi-treated and NaIO3-insulted cells (K-O) were compared to untreated (A-E) and untreated and NaIO3-insulted cells (F-J). With an estimated 196 million people suffering from age-related macular degeneration (AMD) in 2020 and predicted to increase to 288 million by 2040,1 dry AMD, representing 70%-90% of AMD cases, represents an enormous clinical need with no current therapies. [Extracted from the article]