Gene therapy aims at intercellular delivery of functional genetic material to
the affected area to restore its function or block a dysfunctional gene using viral vectors
(Adeno-associated virus) or non-viral vectors (liposomes, SLNs). Gene therapy for the
management of ocular diseases is emerging with improved and encouraging results.
The Eye has well-defined anatomy, tight ocular barriers, and immune-privileged. It is a
perfect target for gene therapy. Recently, many clinical trials are underway or have
been completed. The success of these clinical trials promotes the treatment of several
ocular diseases (Age-related macular degeneration, glaucoma, retinitis pigmentosa, and
choroideremia). Gene therapy should possess an efficient targeting capacity and
longstanding gene expression. Viral vectors are mainly used for gene therapy, but due
to the risk associated with immunogenicity and mutagenesis, non-viral vectors are
widely utilized. This chapter summarizes the recent development of therapeutic gene
delivery approaches for the effective management of ocular diseases and their use in
ophthalmology.
Keywords: Gene therapy, Non-viral, Ocular delivery, Ocular gene therapy, Viral.
