AI Article Synopsis
- Voretigene neparvovec-rzyl is a newly approved gene therapy for Leber congenital amaurosis, reflecting a growing interest in using gene therapy for eye diseases.
- The eye's unique features, like immune privilege and reduced size, make it an ideal candidate for targeted gene therapy using adeno-associated virus (AAV) vectors.
- The review highlights ongoing AAV trials for various eye diseases, including both genetic and acquired disorders, while emphasizing the need for careful vector design in treating more common conditions like age-related macular degeneration and glaucoma.
Article Abstract
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.
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| http://dx.doi.org/10.1111/ceo.13416 | DOI Listing |
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