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Article

Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells

9 Mar 2021

Abstract excerpt

Dry age-related macular degeneration (AMD) is characterised by loss of central vision and currently has no approved medical treatment. Dysregulation of the complement system is thought to play an important role in disease pathology and supplementation of Complement Factor I (CFI), a key regulator of the complement system, has the potential to provide a treatment option for AMD. In this study, we demonstrate the...

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PMID: 33750925
DOI: 10.1038/s41434-021-00239-9

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