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Gene therapy and the adeno‐associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations
Author(s) -
RamloganSteel Charmaine A.,
Murali Aparna,
Andrzejewski Slawomir,
Dhungel Bijay,
Steel Jason C.,
Layton Christopher J.
Publication year - 2019
Publication title -
clinical and experimental ophthalmology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.3
H-Index - 74
eISSN - 1442-9071
pISSN - 1442-6404
DOI - 10.1111/ceo.13416
Subject(s) - medicine , genetic enhancement , adeno associated virus , disease , clinical trial , macular degeneration , gene delivery , glaucoma , intensive care medicine , bioinformatics , vector (molecular biology) , gene , ophthalmology , pathology , genetics , biology , recombinant dna
Voretigene neparvovec‐rzyl was recently approved for the treatment of Leber congenital amaurosis, and the use of gene therapy for eye disease is attracting even greater interest. The eye has immune privileged status, is easily accessible, requires a reduced dosage of therapy due to its size and is highly compartmentalized, significantly reducing systemic spread. Adeno‐associated virus (AAV), with its low pathogenicity, prolonged expression profile and ability to transduce multiple cell types, has become the leading gene therapy vector. Target diseases have moved beyond currently untreatable inherited dystrophies to common, partially treatable acquired conditions such as exudative age‐related macular degeneration and glaucoma, but use of the technology in these conditions imposes added obligations for caution in vector design. This review discusses the current status of AAV gene therapy trials in genetic and acquired ocular diseases, and explores new scientific developments, which could help ensure effective and safe use of the therapy in the future.