Adeno-Associated Virus Mediated Gene Therapy for Corneal Diseases

• Main Authors: Prabhakar Bastola, Liujiang Song, Brian C. Gilger, Matthew L. Hirsch
• Format: Article
• Language: English
• Published: MDPI AG 2020-08-01
• Series: Pharmaceutics
• Subjects: adeno-associated viruses; cornea; corneal diseases; AAV gene therapy; rAAV
• Online Access: https://www.mdpi.com/1999-4923/12/8/767

<b> </b>According to the World Health Organization, corneal diseases are the fourth leading cause of blindness worldwide accounting for 5.1% of all ocular deficiencies. Current therapies for corneal diseases, which include eye drops, oral medications, corrective surgeries, and corneal transplantation are largely inadequate, have undesirable side effects including blindness, and can require life-long applications. Adeno-associated virus (AAV) mediated gene therapy is an optimistic strategy that involves the delivery of genetic material to target human diseases through gene augmentation, gene deletion, and/or gene editing. With two therapies already approved by the United States Food and Drug Administration and 200 ongoing clinical trials, recombinant AAV (rAAV) has emerged as the in vivo viral vector-of-choice to deliver genetic material to target human diseases. Likewise, the relative ease of applications through targeted delivery and its compartmental nature makes the cornea an enticing tissue for AAV mediated gene therapy applications. This current review seeks to summarize the development of AAV gene therapy, highlight preclinical efficacy studies, and discuss potential applications and challenges of this technology for targeting corneal diseases.