Open AccessAAV and Compacted DNA Nanoparticles for the Treatment of Retinal Disorders: Challenges and Future Prospects
Author(s) -
Zongchao Han,
Shan M. Conley,
Muna I. Naash
Publication year - 2011
Publication title -
investigative ophthalmology and visual science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.935
H-Index - 218
eISSN - 1552-5783
pISSN - 0146-0404
DOI - 10.1167/iovs.10-6916
Subject(s) - gene delivery , genetic enhancement , vector (molecular biology) , viral vector , clinical trial , computational biology , medicine , bioinformatics , risk analysis (engineering) , biology , gene , genetics , recombinant dna
Gene therapy based on delivery of viral and nonviral vectors has shown great promise for the treatment of human ocular diseases; however, limitations have consistently prevented its widespread clinical application. Viral vectors have generally been better in terms of efficiency but have safety concerns. Nonviral vectors, on the other hand, offer safety but have often been disappointing in terms of efficiency of nuclear delivery and gene expression. Extensive animal studies have reported significant progress using both systems, but thus far only a few studies have shown promise in human clinical trials. This article reviews both viral and nonviral work with focus on two candidates for clinical ocular application--AAV and nanoparticles. Of particular interest are various requirements for successful clinical application of these technologies including vector trafficking, delivery, specific gene expression, and treatment safety, and tolerance.