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Truncated Complement Factor H Y402 Gene Therapy Cures C3 Glomerulonephritis

2024-09-21

Abstract excerpt

Patients with both age-related macular degeneration (AMD) and C3 glomerulonephritis (C3G) are challenged by the absence of effective therapies to reverse and eliminate their disease burden. Capitalizing on complement dysregulation as both a significant risk factor for AMD and the known pathophysiology of C3G, we investigated the potential for adeno-associated virus (AAV) delivery of complement factor H (CFH) to re...

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Literature Corpus work
1f1be49c-02a3-5f68-a3ef-750aaeb1543a
DOI
10.1101/2024.09.17.613471
Open publication

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Truncated Complement Factor H Y402 Gene Therapy Cures C3 GlomerulonephritisDOI 10.1101/2024.09.17.613471
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