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1. Schnabolk G, Parsons N, Obert E, Annamalai B, Nasarre C, Tomlinson S, Lewin AS, Rohrer B: Delivery of CR2-fH Using AAV Vector Therapy as Treatment Strategy in the Mouse Model of Choroidal Neovascularization. Mol Ther Methods Clin Dev; 2018 Jun 15;9:1-11

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Delivery of CR2-fH Using AAV Vector Therapy as Treatment Strategy in the Mouse Model of Choroidal Neovascularization.
  • Complement activation plays a significant role in age-related macular degeneration (AMD) pathogenesis, and polymorphisms interfering with factor H (fH) function, a complement alternative pathway (AP) inhibitor, are associated with increased AMD risk.
  • We have previously validated an AP inhibitor, a fusion protein consisting of a complement receptor 2 fragment linked to the inhibitory domain of fH (CR2-fH) as an efficacious treatment for choroidal neovascularization (CNV) when delivered intravenously.
  • Here we tested an alternative approach of AAV-mediated delivery (AAV5-VMD2-CR2-fH or AAV5-VMD2-mCherry) using subretinal delivery in C57BL/6J mice.
  • Secretion of CR2-fH was confirmed in polarized retinal pigment epithelium (RPE) cells.
  • A safe concentration of AAV5-VMD2-CR2-fH was identified using electroretinography, optical coherence tomography (OCT), RPE morphology, and antibody profiling.
  • One month after gene delivery, CNV was induced using argon laser photocoagulation.
  • OCT assessment demonstrated reduced CNV with AAV5-VMD2-CR2-fH administration.
  • Bioavailability studies revealed that gene-therapy delivered similar levels of CR2-fH to the RPE/choroid as treatment by intravenous injections, and C3a ELISA verified reduced CNV-associated ocular C3a production.
  • These results contribute to existing data illustrating the importance of the AP of complement in CNV development and its potential role in AMD treatment.
  • Demonstration of AAV-vector efficacy opens new avenues for the development of treatment strategies.

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  • (PMID = 29234687.001).
  • [ISSN] 2329-0501
  • [Journal-full-title] Molecular therapy. Methods & clinical development
  • [ISO-abbreviation] Mol Ther Methods Clin Dev
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Keywords] NOTNLM ; AAV / age-related macular degeneration / choroidal neovascularization / complement factor H / retinal pigment epithelium
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