Nanoparticles and Nanocarriers Based Pharmaceutical Formulations

Gene Therapy: A New Avenue for the Management of Ophthalmic Diseases

Author(s): Kesavan Karthikeyan*, Nivedita Gautam, Olivia Parra-Marín and Selvasankar Murugesan

Pp: 395-435 (41)

DOI: 10.2174/9789815049787122010016

* (Excluding Mailing and Handling)


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.

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