Find all
associated with

  Your query has expired (error code 641534678594).
Refine your query (more in Advanced-Search):
 Search all of MEDLINE   Focus on the recent 5 years   Focus on the current year   Focus on the last 30 days   More choices ...
 Focus on articles with free fulltexts   More choices ...
 Do simple 'keyword' search (no query expansion)

[X] Close
You are about to erase all the values you have customized, search history, page format, etc.
Click here to RESET all values       Click here to GO BACK without resetting any value
Item 1 of about 1
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.

  • [Email] Email this result item
    Email the results to the following email address:   [X] Close
  • (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
  •  go-up   go-down